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For example, on November 3, 2025, we announced topline results from our ESSENCE trial, a confirmatory trial intended to verify the clinical benefits of VYONDYS 53 and AMONDYS 45, which primary endpoint did not meet statistical significance.
−Removed: We submitted supplemental new drug applications ("sNDAs") related to these products in April 2026.
+Added: We submitted sNDAs related to these products in April 2026, which the FDA accepted for filing in June 2026.
However, these results could lead to regulatory actions from the FDA, including changes to our drug labels, revocation of accelerated approvals and directives to remove these products from the market altogether.
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Recent years have seen a number of reform initiatives focused on drug pricing and payment.
−Removed: For example, the Inflation Reduction Act ("IRA"), passed in 2022, IRA has had and will likely continue to have a significant impact on the pharmaceutical industry.
+Added: For example, the Inflation Reduction Act ("IRA"), passed in 2022, has had and will likely continue to have a significant impact on the pharmaceutical industry.
In 2025, the current presidential administration issued two executive orders with multiple directives aimed at lowering drug prices.
In the wake of these executive orders and related executive initiatives, a number of pharmaceutical manufacturers have announced direct-to-consumer offerings with discounted prices and/or reached agreement with the federal government regarding pricing for drugs, including prices for Medicaid drugs and newly launched products.
+Added: In January 2026, the current presidential administration announced a healthcare plan calling for codification of most-favored-nation pricing arrangements with pharmaceutical manufacturers, and in April 2026, the administration issued a further executive order establishing tariffs on certain imported patented pharmaceuticals while offering relief from such tariffs to manufacturers that commit to most-favored-nation pricing and to onshoring research and manufacturing operations.
+Added: The federal government has also launched a website that offers access to pharmaceutical direct-to-consumer discount drug offers and discloses generic drug pricing.
Many of these reform initiatives would require additional legal and/or administrative action to implement and may be subject to legal challenge.
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Healthcare and Other Reform ." There is no assurance that federal or state health care reform will not adversely affect our future business and financial results, and we cannot predict how future federal or state legislative, judicial or administrative changes relating to healthcare policy will affect our business.
−Removed: government, state legislatures and foreign governments have shown significant interest in implementing cost-containment programs to limit the growth
−Removed: of government-paid and private insurance healthcare costs, including proposed or implemented reforms.
+Added: government, state legislatures and foreign governments have shown significant interest in implementing cost-containment programs to limit the growth of government-paid and private insurance healthcare costs, including proposed or implemented reforms.
Cost containment initiatives might include, among other possible actions, implementation or modification of:
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Raimondo, the U.S.
−Removed: Supreme Court overruled the “Chevron doctrine,” which gives deference to regulatory agencies’ statutory interpretations in litigation against federal government agencies, such as the FDA, the Centers for Medicare & Medicaid Services (“CMS”) and other federal agencies where the law is ambiguous.
+Added: Supreme Court overruled the “Chevron doctrine,” which gives deference to regulatory agencies’ statutory interpretations in litigation against federal government agencies, such as the FDA, the Centers for Medicare & Medicaid Services (the “CMS”) and other federal agencies where the law is ambiguous.
The Loper decision could result in additional legal challenges to regulations and guidance issued by federal agencies, including the FDA and the CMS, on which we rely.
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Additionally, in November 2025, we announced topline results from our ESSENCE trial, a confirmatory trial intended to verify the clinical benefits of VYONDYS 53 and AMONDYS 45, which primary endpoint did not meet statistical significance.
−Removed: We submitted sNDAs related to these products in April 2026.
+Added: We submitted sNDAs related to these products in April 2026, which the FDA accepted for filing in June 2026.
However, these results could lead to regulatory actions from the FDA, including changes to our drug labels or revocation of accelerated approvals and directives to remove these products from the market altogether, negatively impact patient demand for these products or result in changes to reimbursement and coverage of these products by payors.
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Public perception may be influenced by claims that gene therapy is unsafe, and gene therapy may not gain the acceptance of the public or the medical community.
−Removed: In particular, our success will depend upon physicians who specialize in the treatment of genetic diseases targeted by our product candidates, prescribing treatments that involve the use of our product candidates in lieu of, or in addition to, existing treatments with which they are familiar and for which greater clinical data may be available.
+Added: In particular, our success will depend upon physicians who
+Added: specialize in the treatment of genetic diseases targeted by our product candidates, prescribing treatments that involve the use of our product candidates in lieu of, or in addition to, existing treatments with which they are familiar and for which greater clinical data may be available.
In addition, ethical, social and legal concerns about gene therapy, genetic testing and genetic research could result in additional regulations or prohibiting the processes we may use.
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We may not receive approval to commercialize these products in additional countries.
−Removed: Our partner for ELEVIDYS, Roche, has received certain approvals for ELEVIDYS in territories outside of the U.S.
In November 2016, we submitted a MAA for eteplirsen to the EMA and the application was validated in December 2016.
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On September 24, 2025, the EC refused marketing authorization under Regulation (EC) No 726/2004 of the European Parliament and of the Council for ELEVIDYS for ambulatory individuals aged three to seven years with Duchenne.
−Removed: We also announced in June 2025 that we paused our ENVISION study for ELEVIDYS and such study remains paused.
+Added: We also announced in June 2025 that we paused our ENVISION study for ELEVIDYS, and such study remains on clinical hold.
In order to market any product in a country outside of the U.S., we must comply with numerous and varying regulatory requirements for approval in those countries regarding demonstration of evidence of the product’s safety and efficacy and governing, among other things, labeling, distribution, advertising, and promotion, as well as pricing and reimbursement of the product.
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Even if a healthcare provider is interested in obtaining access to our products for its patient through our EAP, the patient may not be able to obtain access to our products if funding for the drug is not secured.
−Removed: Also geo-political changes and challenges might negatively impinge upon future revenue generated through our EAP.
+Added: Also geo-political and distribution changes and challenges might negatively impinge upon future revenue generated through our EAP.
To date, our business and financial results have not yet been materially adversely affected by the ongoing conflict between Russia and Ukraine, recent events in Venezuela, or the conflict in the Middle-East.
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could disrupt operations and have a negative impact on our business.
−Removed: Any failure to maintain revenues from sales of our products through our EAP and/or to generate revenues from commercial sales of these products exceeding historical sales due to geo-political challenges like those potentially resulting from the ongoing conflict between Russia and Ukraine or the instability in the Middle-East or Venezuela , could have a material adverse effect on our business, financial condition, results of operations and growth prospects.
+Added: Any failure to maintain revenues from sales of our products through our EAP and/or to generate revenues from commercial sales of these products exceeding historical sales due to distribution or geo-political challenges, including those potentially resulting from the ongoing conflict between Russia and Ukraine or the instability in the Middle-East or Venezuela , could have a material adverse effect on our business, financial condition, results of operations and growth prospects.
Failure to obtain or maintain regulatory exclusivity for our products could result in our inability to protect our products from competition and our business may be adversely impacted.
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Implementation and enforcement of regulatory exclusivity, which may consist of regulatory data protection and market protection, varies widely from country to country.
−Removed: Failure to qualify for regulatory exclusivity, or failure to obtain or maintain the extent or duration of such protections that we expect in each of the markets for our products due to challenges, changes or
−Removed: interpretations in the law or otherwise, could affect our revenues for our products or our decision on whether to market our products in a particular country or countries or could otherwise have an adverse impact on our results of operations.
+Added: Failure to qualify for regulatory exclusivity, or failure to obtain or maintain the extent or duration of such protections that we expect in each of the markets for our products due to challenges, changes or interpretations in the law or otherwise, could affect our revenues for our products or our decision on whether to market our products in a particular country or countries or could otherwise have an adverse impact on our results of operations.
We are not guaranteed to receive or maintain regulatory exclusivity for our current or future products, and if our products that are granted orphan status were to lose their status as orphan drugs or the data or marketing exclusivity provided for orphan drugs, our business and operations could be adversely affected.
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For example, the exclusivity period for EXONDYS 51, which received initial FDA approval in September 2016, ended in September 2023.
−Removed: Recent litigation has raised questions about the appropriate scope of orphan drug exclusivity.
−Removed: A decision in 2021 by the U.S.
−Removed: Court of Appeals for the Eleventh Circuit in Catalyst Pharmaceuticals, Inc.
−Removed: Becerra regarding interpretation of the Orphan Drug Act’s exclusivity provisions as applied to drugs and biologics approved for orphan indications narrower than the product’s orphan designation has the potential to significantly broaden the scope of orphan exclusivity for such products.
−Removed: Specifically, the court held that, under the statute, orphan drug exclusivity blocks approval of another company’s application for the same chemical or biological product for the entire disease or condition for which the drug is granted orphan drug designation, regardless of whether the ultimate marketing approval only covered a narrower use or indication.
−Removed: While the FDA has since taken the position that it will continue to apply orphan drug exclusivity only on the basis of the specific indication, the Supreme Court’s recent decision in 2024 in Loper Bright Enterprises v.
−Removed: Raimondo has the potential to impact how the Agency applies the Catalyst decision.
−Removed: Our ability to obtain or seek to work around orphan exclusivity, as well as our ability to retain orphan exclusivity that the FDA previously has recognized for our products, may be impacted depending on how the Catalyst decision is ultimately implemented.
−Removed: Legislation has been introduced to amend the Orphan Drug Act in a way that may prevent these effects of the Catalyst decision, but it is unclear if or when such legislation could be enacted.
In addition, we may face risks with maintaining regulatory exclusivities for our products, and our protection may be circumvented, even if maintained.
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However, there is considerable uncertainty as to the interpretation of these guidelines.
−Removed: As illustrated by this guidance, orphan drug exclusivity as applied to gene therapy products is an evolving area subject to change and interpretation by the FDA and therefore, we cannot be certain as to how the FDA will apply those rules to ELEVIDYS, gene therapy
−Removed: product candidates or our siRNA programs.
−Removed: Similarly, pursuant to the 2018 Commission Regulation, two gene therapy medicinal products are not considered similar when there are differences in the therapeutic sequence, viral vector, transfer system, regulatory sequences or manufacturing technology that significantly affect the biological characteristics and/or biological activity relevant for the intended therapeutic effect and/or safety attributes of the product.
+Added: As illustrated by this guidance, orphan drug exclusivity as applied to gene therapy products is an evolving area subject to change and interpretation by the FDA and therefore, we cannot be certain as to how the FDA will apply those rules to ELEVIDYS, gene therapy product candidates or our siRNA programs.
+Added: Similarly, pursuant to the 2018 Commission Regulation, two gene therapy medicinal
+Added: products are not considered similar when there are differences in the therapeutic sequence, viral vector, transfer system, regulatory sequences or manufacturing technology that significantly affect the biological characteristics and/or biological activity relevant for the intended therapeutic effect and/or safety attributes of the product.
If we are unable to successfully maintain and further develop internal commercialization capabilities, sales of our products may be negatively impacted.
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For example, we face competition in the fields of Duchenne, gene therapy and siRNA by third parties who are developing or who had once developed:
−Removed: (i) exon skipping product candidates, such as Wave (targeting various exons, including 53 and 51), Nippon (targeting various exons, including 51 and 45, and notably for exon 53 for which it has received accelerated FDA approval for its product Viltepso (viltolarsen)), Dyne pursuing antibody-oligonucleotide conjugates for exons 44, 45, 51, and 53, Novartis (formerly Avidity Biosciences ("Avidity")) pursuing antibody-oligonucleotide conjugates for exons 44, 45 and 51, SQY Therapeutics and BioMarin (for exon 51), Entrada (notably for exon 44, 45, 50, and 51);
+Added: (i) exon skipping product candidates, such as Wave (targeting various exons, including 53);
+Added: Nippon (targeting various exons, including 51 and 44, and notably for exon 53 for which it has received accelerated FDA approval for its product Viltepso (viltolarsen));
+Added: Dyne pursuing antibody-oligonucleotide conjugates for exons 44, 45, 51 (for which the FDA has accepted for review a BLA), 53, and 55;
+Added: Novartis (formerly Avidity Biosciences ("Avidity")) pursuing antibody-oligonucleotide conjugates for exons 44, 45 and 51, SQY Therapeutics and BioMarin (for exon 51);
+Added: and Entrada (notably for exon 44, 45, 50, and 51);
(ii) gene therapies, such as Genethon and Solid (also in partnership with Ultragenyx), Regenxbio and Insmed;
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(v) other approaches that may be palliative in nature or potentially complementary with our products and product candidates and that are or were once being developed including but not limited to, Santhera (approved product vamorolone), Capricor Therapeutics (in partnership with Nippon), BioPhytis, Italfarmaco (approved product Givinostat), Dystrogen and Edgewise Therapeutics.
−Removed: Although BioMarin announced on May 31, 2016 its intent to discontinue clinical and regulatory development of drisapersen as well as its other clinical stage candidates, BMN 044, BMN 045 and BMN 053, then-currently in Phase 2 studies for distinct forms of Duchenne, it further announced its intent to continue to explore the development of next generation oligonucleotides for the treatment of Duchenne.
−Removed: Indeed, BioMarin is conducting clinical trials for BMN-351, an oligonucleotide therapy.
−Removed: In addition, while Wave announced its intention to discontinue development of suvodirsen and suspend development of WVE-N531, it is conducting clinical trials for its exon 53 oligonucleotide, WVE-N531.
−Removed: In the siRNA field, we face competition by third parties who are also developing product candidates targeting the same disease states as our product candidates, including but not limited to Novartis (formerly Avidity) (DM1, FSHD), Dyne (DM1, FSHD), Arthex Biotech (DM1), PepGen (DM1), Vertex Pharmaceuticals ("Vertex") (DM1), Entrada (FSHD), Celularity (FSHD), EpiCrispr Biotechnologies (FSHD), Biohaven (SCA), Vico Therapeutics (SCA, Huntington’s), Skyhawk Therapeutics (SCA, Huntington’s), uniQure (Huntington’s), Roche Ionis (Huntington’s), Wave (Huntington’s), and PTC (Huntington’s).
+Added: In the siRNA field, we face competition by third parties who are also developing product candidates targeting the same disease states as our product candidates, including but not limited to Novartis (formerly Avidity) (DM1, FSHD), Dyne (DM1, FSHD), Arthex Biotech (DM1), PepGen (DM1), Vertex Pharmaceuticals ("Vertex") and Entrada (DM1), Celularity (FSHD), EpiCrispr Biotechnologies (FSHD), Biohaven (SCA), Vico Therapeutics (SCA, Huntington’s), Skyhawk Therapeutics (SCA, Huntington’s), uniQure (Huntington’s), Wave (Huntington’s), and PTC (Huntington’s).
Some, but not all, of these entities’ product candidates use RNA technologies.
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Because many of our products are in various stages of preclinical and clinical development, and given the unpredictability inherent in drug development, it is difficult to predict which third parties may provide the most competition.
−Removed: If any of our competitors are successful in obtaining regulatory approval for any of their product candidates, it may limit our ability to enter into the market, gain market share or maintain market share in the Duchenne, DM1, FSHD, SCA, Huntington's, and IPF spaces or other diseases targeted by our platform technologies, products and product candidate pipeline.
+Added: If any of our competitors are successful in obtaining regulatory approval for any of their product candidates, it may limit our ability to enter into the market, gain market share or maintain market share in the Duchenne, DM1, FSHD, SCA, and Huntington's spaces or other diseases targeted by our platform technologies, products and product candidate pipeline.
It is possible that our competitors will succeed in developing technologies that, in addition to limiting the market size for our products or product candidates, impact the regulatory approval and post-marketing process for our products and product candidates, are more effective than our products or product candidates or would render our technologies obsolete or noncompetitive.
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• collaborators may not properly maintain or defend our intellectual property rights or may use our intellectual property or proprietary information in a way that gives rise to actual or threatened litigation that could jeopardize or invalidate our intellectual property or proprietary information or expose us to potential liability;
−Removed: • disputes may arise between us and a collaborator that cause the delay or termination of the research, development or commercialization of our products or product candidates, or that result in costly litigation or arbitration that diverts management attention and resources;
+Added: • disputes may arise between us and a collaborator or strategic partner that cause the delay or termination of the research, development, manufacturing, or commercialization of our products or product candidates, or that result in costly litigation or arbitration that diverts management attention and resources;
• collaborations may be terminated and, if terminated, may eliminate our rights to commercialize certain product candidates or may result in a need for additional capital;
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Roche’s development and commercialization activities in the territories where it is the lead party may adversely impact our own efforts in the U.S.
−Removed: As a further example, Arrowhead conducted the Phase 1/2 single ascending dose studies for SRP-1001 (FSHD1) and SRP-1003 (DM1), for which we announced early results in March 2026.
+Added: As a further example, Arrowhead has conducted the Phase 1/2 ascending dose studies for SRP-1001 (FSHD1) and SRP-1003 (DM1) to date, for which we announced early and limited results in March 2026.
Failure by Roche or Arrowhead to meet their obligations under the Roche Collaboration Agreement and Arrowhead Collaboration Agreement, to apply sufficient efforts at developing and commercializing collaboration products, or to comply with applicable legal or regulatory requirements, may materially adversely affect our business and our results of operations.
In addition, to the extent we rely on Roche to commercialize any products for which we obtain regulatory approval, we will receive less revenues than if we commercialized these products ourselves.
−Removed: Even if we achieve the long-term benefits associated with strategic transactions, our expenses and short-term costs may increase materially and adversely affect our liquidity and short-term net income (loss).
+Added: Even if we achieve the long-term benefits associated with strategic transactions, our expenses and short-term costs may increase materially and adversely affect our liquidity and short-term net (loss) income.
Future licenses or acquisitions could result in potentially dilutive issuances of our equity securities, the incurrence of debt, the creation of contingent liabilities, impairment or expenses related to goodwill, and impairment or amortization expenses related to other intangible assets, which could harm our financial condition.
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Successful completion of clinical trials at each applicable stage of development is a prerequisite to submitting a marketing application to the regulatory agencies and, consequently, the ultimate approval and commercial marketing of any of our product candidates for the indications in which we develop them.
−Removed: We do not know whether any of our clinical trials, or those with our strategic partners, will begin or be completed, and results announced, as planned or expected, if at all, as the commencement and completion of clinical trials and announcement of results is often delayed or prevented for a number of reasons, including, among others:
+Added: We do not know whether any of our clinical trials, or those with our strategic
+Added: partners, will begin or be completed, and results announced, as planned or expected, if at all, as the commencement and completion of clinical trials and announcement of results is often delayed or prevented for a number of reasons, including, among others:
• denial by the regulatory agencies of permission to proceed with our planned clinical trials or any other clinical trials we may initiate, or placement of a clinical trial on hold.
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• the recruitment and retention of employees, consultants or contractors with the required level of expertise.
−Removed: Further, any reduction in the FDA’s workforce could delay or materially impact the FDA’s feedback on our development programs, including through meetings and other informal interactions, and affect the FDA’s review and oversight of our product
+Added: Further, any reduction in the FDA’s workforce could delay or materially impact the FDA’s feedback on our development programs, including through meetings and other informal interactions, and affect the FDA’s review and oversight of our product candidates.
Additionally, changes in the FDA personnel under the current presidential administration may lead to changes in the regulations, policies and operations of the FDA, which may impact our clinical development plans.
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In addition, as a result of any government shutdown, the FDA staff may be unable to process and review regulatory submissions in a timely manner or at all.
−Removed: Any inability to complete successfully pre-clinical and clinical development could result in additional costs to us or impair our ability to generate revenues from product sales, regulatory and commercialization milestones and royalties, as well as our ability to maintain our accelerated approvals.
+Added: Any inability to complete successfully pre-clinical and clinical development could result in additional costs to us or impair our ability to generate revenues from product sales, regulatory and commercialization milestones and royalties, as well as our ability
+Added: to maintain our accelerated approvals.
In addition, manufacturing or formulation changes to our product candidates often require additional studies to demonstrate comparability of the modified product candidates to earlier versions.
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For example, announcements for SRP-9003 include:
−Removed: in March 2022, we announced 24-month functional data from two clinical trial participants in the high-dose cohort, and 36-month functional data from three clinical trial participants in the low-dose cohort for SRP-9003.
+Added: in March 2022, we announced 24-month functional data from two clinical trial participants in the high-dose cohort, and 36-month functional data from
+Added: three clinical trial participants in the low-dose cohort for SRP-9003.
In September 2025 we presented 18 month functional data compared to external control for 6 ambulant patients from the SRP-9003-101 study and 5 non-ambulant patients from the SRP-9003-102 study, and 5 year safety data from the SRP-9003-101 study.
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to non-ambulatory patients in June 2025.
−Removed: Additionally, in July 2025, we disclosed a reported case of ALF in a non-ambulatory patient participating in our Stage 1/2 LGMD trial for SRP-9004, who was not treated with ELEVIDYS.
+Added: Additionally, in July 2025, we disclosed a reported case of ALF in a non-ambulatory patient participating in our Phase 1 LGMD trial for SRP-9004, who was not treated with ELEVIDYS.
Thereafter, in response to a request from the FDA that we voluntarily stop all shipments of ELEVIDYS in the U.S., we suspended shipment of ELEVIDYS in the U.S.
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The CBER works closely with the National Institutes of Health (the “NIH”).
−Removed: The FDA and the NIH have published guidance documents with respect to the development and submission of gene therapy protocols.
−Removed: For example, on January 28, 2020, the FDA issued final guidance documents that updated draft guidance documents that were originally released in July 2018 to reflect recent advances in the field, and to set forth the framework for the development, review and approval of gene therapies.
−Removed: These final guidance documents pertain to the development of gene therapies for the treatment of specific disease categories, including rare diseases, and to manufacturing and long-term follow up
−Removed: issues relevant to gene therapy, among other topics.
−Removed: The FDA also issued a new guidance document in September 2021 describing the FDA’s approach for determining whether two gene therapy products were the same or different for the purpose of assessing orphan drug exclusivity, as well as a final guidance document in January 2024 on human gene therapy product incorporating human genome editing.
−Removed: The FDA also issued a draft guidance in December 2023 that provides recommendations for developing a potency assurance strategy for gene therapy products.
−Removed: In addition, the FDA can put an IND on hold if the information in an IND is not sufficient to assess the risks in pediatric patients.
−Removed: These regulatory review agencies, committees and advisory groups and the new requirements and guidelines they promulgate may lengthen the regulatory review process, require us to perform additional or larger studies, increase our development costs, lead to changes in regulatory positions and interpretations, delay or prevent approval and commercialization of these treatment candidates or lead to significant post-approval studies, limitations or restrictions.
+Added: The FDA has published guidance documents in recent years with respect to the regulation of gene therapy products.
+Added: These guidance documents have generally been intended by FDA to promote greater transparency and increase efficiency in the development of gene therapy products.
+Added: Nevertheless, there can be no assurance that these guidance documents will enable the accelerated development or approval of any particular gene therapy products.
+Added: These regulatory review agencies, committees and advisory groups, including changes in FDA leadership, and the new requirements and guidelines they promulgate may lengthen the regulatory review process, require us to perform additional or larger studies, increase our development costs, lead to changes in regulatory positions and interpretations, delay or prevent approval and commercialization of these treatment candidates or lead to significant post-approval studies, limitations or restrictions.
As we advance our product candidates, we will be required to consult with these regulatory and advisory groups and comply with applicable requirements and guidelines, failure of which may lead to delayed or discontinued development of our product candidates.
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The FDA may interpret the data from these trials differently than us and, regardless of the trial results, not permit us to resume shipments to non-ambulatory patients.
+Added: In addition, in November 2025, we announced topline results from our ESSENCE trial, a confirmatory trial intended to verify the clinical benefits of VYONDYS 53 and AMONDYS 45, which primary endpoint did not meet statistical significance.
+Added: We submitted sNDAs related to these products in April 2026, which the FDA accepted for filing in June 2026.
+Added: The FDA may interpret the results of the ESSENCE trial differently from us, which could lead to regulatory actions from the FDA, including changes to our drug labels, revocation of accelerated approvals and directives to remove these products from the market altogether.
If our study data do not consistently or sufficiently demonstrate the safety or efficacy of any of our product candidates, the regulatory approvals for such product candidates could be significantly delayed as we work to meet approval requirements, or, if we are not able to meet these requirements, such approvals could be withheld or withdrawn.
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A fast track product designation is designed to facilitate the clinical development and expedite the review of drugs intended to treat a serious or life-threatening condition which demonstrate the potential to address an unmet medical need.
−Removed: A breakthrough therapy is defined as a drug that is intended, alone or in
−Removed: combination with one or more other drugs, to treat a serious or life-threatening disease or condition, where preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.
+Added: A breakthrough therapy is defined as a drug that is intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition, where preliminary clinical
+Added: evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.
A RMAT designation is designed to accelerate approval for regenerative advanced therapies such as our gene therapy product candidates.
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For example, we announced topline results from our ESSENCE trial, a confirmatory trial intended to verify the clinical benefits of VYONDYS 53 and AMONDYS 45.
−Removed: We submitted sNDAs related to these products in April 2026.
+Added: We submitted sNDAs related to these products in April 2026, which the FDA accepted for filing in June 2026.
From time to time, we also may disclose interim data from our preclinical studies and clinical trials.
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With respect to the pre-commercial distribution of our products to patients outside of the U.S., we have contracted with third party distributors and service providers to distribute our products in certain countries through our EAP.
−Removed: We will need to continue building out our network for commercial distribution in jurisdictions in which our products are approved, which will also require third party contracts.
−Removed: The use of distributors and service providers involves certain risks, including, but not limited to, risks that these organizations will not comply with applicable laws and regulations, or not provide us with accurate or timely information regarding serious adverse events and/or product complaints regarding our products.
+Added: We will need to maintain and continue building out our network for commercial distribution in jurisdictions in which our products are approved, as well as in jurisdictions in which our products are available through our EAP, all of which will require third party contracts.
+Added: The use of distributors and service providers involves certain risks, including, but not limited to, risks that these organizations will not comply with contractual obligations and applicable laws and regulations, or not provide us with accurate or timely information regarding serious adverse events and/or product complaints regarding our products.
Any such events may result in regulatory actions that may include suspension or termination of the distribution and sale of our products in a certain country, loss of revenue, and/or reputational damage, which could harm our results of operations and business.
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Our reliance on third parties requires us to share our proprietary information, which increases the possibility that a competitor will discover them or that our proprietary information will be misappropriated or inadvertently disclosed.
−Removed: Our reliance on third-party collaborators requires us to disclose our proprietary information to these parties, which could increase the risk that a competitor will discover this information or that this information will be misappropriated or disclosed without
−Removed: our intent to do so.
+Added: Our reliance on third-party collaborators requires us to disclose our proprietary information to these parties, which could increase the risk that a competitor will discover this information or that this information will be misappropriated or disclosed without our intent to do so.
If any of these events were to occur, then our ability to obtain patent protection or other intellectual property rights could be irrevocably jeopardized, and costly, distracting litigation could ensue.
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Our dependence on these parties, including failure on our part to accurately anticipate product demand and timely secure manufacturing capacity to meet commercial, EAP, clinical and pre-clinical product demand may impair the availability of product for commercial supply or to successfully support various programs, including research and development and the potential commercialization of additional product candidates in our pipeline.
−Removed: We rely on, and expect to continue relying on for the foreseeable future, a limited number of third parties to manufacture and supply materials (including raw materials, starting materials and subunits), API and drug product and to provide labeling and packaging of vials and storage of our products and product candidates.
+Added: We rely on, and expect to continue relying on for the foreseeable future, a limited number of third parties to manufacture and supply materials (including raw materials, starting materials and subunits), API and drug product and to provide release testing and labeling and packaging of vials and storage of our products and product candidates.
The limited number of third parties with facilities, expertise and the capability suited to manufacture our products and product candidates creates a risk that we may not be able to obtain materials and APIs in the quantity and purity that we require.
−Removed: As of the date of this Quarterly Report, we have dual sourcing for the APIs and drug product for all three of our PMO commercial products and one source for ELEVIDYS drug substance and drug product manufacturing.
+Added: As of the date of this Quarterly Report, we have dual sourcing for the APIs for all three of our PMO commercial products and EXONDYS 51 drug product, one active source for AMONDYS 45 and VYONDYS 53 drug product, and one source for ELEVIDYS drug substance and drug product manufacturing.
In light of the limited number of third parties with the expertise to produce our products and product candidates, the lead time needed to manufacture them, and the availability of underlying materials, we may not be able to, in a timely manner or at all, establish or maintain sufficient clinical, commercial and other manufacturing arrangements on the commercially reasonable terms necessary to provide adequate supply of our products and product candidates.
Furthermore, we may not be able to obtain the significant financial capital that may be required in connection with such arrangements.
−Removed: Even after successfully engaging third parties to execute the manufacturing process for our products and product candidates, such parties may not comply with the terms and timelines they have agreed to for various reasons, some of which may be out of their or our control, which impacts our ability to execute our business plans on expected or required timelines in connection with the commercialization of our products and the continued development of our product candidates.
+Added: Even after successfully engaging third parties to execute the manufacturing process and release testing for our products and product candidates, such parties may not comply with the terms and timelines they have agreed to for various reasons, some of which may be out of their or our control, which impacts our ability to execute our business plans on expected or required timelines in connection with the commercialization of our products and the continued development of our product candidates.
We may also be impacted by development, operation, or production disruptions involving these third parties and our partners, the reasons for which also may be outside of their or our control.
−Removed: Several factors could cause production interruptions, disturbances or supply chain issues at our third party manufacturing sites, including but not limited to talent acquisition/retention, equipment malfunctions, quality control and quality assurance issues, facility contamination, raw material shortages or contamination, man-made or natural disasters, public-health pandemics or epidemics, disruption in utility services, regulatory decisions and delays and possible negative effects of such delays on supply chains and expected timelines for product availability, production yield issues, shortages of qualified personnel, discontinuation of facility or business, government shutdowns, economic sanctions, human error, or disruptions in the operations of suppliers, including those caused by geopolitical conflicts or tariffs.
−Removed: In addition, the need to prioritize rated orders issued by the Federal Emergency Management Agency pursuant to the U.S.
+Added: Several factors could cause production interruptions, disturbances or supply chain issues at our third party manufacturing, supply, testing, packaging, and distribution sites, including but not limited to talent acquisition/retention, equipment malfunctions, quality control and quality assurance issues, facility contamination, raw material shortages or contamination, man-made or natural disasters, public-health pandemics or epidemics, disruption in utility services, regulatory decisions and delays and possible negative effects of such delays on supply chains and expected timelines for product availability, production yield issues, shortages of qualified personnel, discontinuation of facility or business, government shutdowns, economic sanctions, human error, or disruptions in the operations of suppliers and other partners, including those caused by geopolitical conflicts or tariffs.
+Added: In addition, the need to prioritize rated orders issued by the Federal Emergency Management Agency
+Added: pursuant to the U.S.
Defense Production Act could impact the manufacturing, supply chain and distribution of our products and product candidates.
−Removed: Any interruption in the operation of our third-party manufacturing, supply, or distribution partners or any of their suppliers or partners could result in the cancellation of shipments, loss of product in the manufacturing process or a shortfall in supply of our products, product candidates or materials.
−Removed: In turn, any delay or interruption in the supply of finished commercial or clinical products could hinder our ability to distribute our products to meet
−Removed: commercial or clinical demand or execute our commercialization or clinical trial plans on the timing that we expect, which could result in the loss of potential revenues, adversely affect our ability to gain regulatory or market acceptance, or otherwise adversely affect our business, financial condition and prospects.
−Removed: In addition, several of the components used in our testing are currently from single-source suppliers.
+Added: Any interruption in the operation of our third-party manufacturing, supply, packaging, release testing, or distribution partners or any of their suppliers or partners could result in the cancellation of shipments, loss of product in the manufacturing process or a shortfall in supply of our products, product candidates or materials.
+Added: In turn, any delay or interruption in the supply of finished commercial or clinical products could hinder our ability to distribute our products to meet commercial or clinical demand or execute our commercialization or clinical trial plans on the timing that we expect, which could result in the loss of potential revenues, adversely affect our ability to gain regulatory or market acceptance, or otherwise adversely affect our business, financial condition and prospects.
+Added: In addition, several of the components used in our manufacturing processes and for testing are currently from single-source suppliers.
If these single-source suppliers fail to satisfy the Company's requirements on a timely basis, or if the Company is forced to change suppliers, the Company could suffer delays, a possible loss of revenue, or incur higher costs, any of which could adversely affect its operating results.
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If any of the third parties on which we rely cease providing quality manufacturing and related services to us, and we are not able to engage appropriate replacements in a timely manner, our ability to manufacture our products or product candidates in sufficient quality and quantity required for our planned commercial, pre-clinical and clinical or EAPs, our various product research, development and commercialization efforts would be adversely affected.
−Removed: For example, with respect to ELEVIDYS, we rely on a third party to develop, manufacture, obtain and maintain regulatory approval for necessary diagnostic tests for ELEVIDYS.
−Removed: Any delay or failure by us or our collaborators to develop, obtain or maintain regulatory approval of the necessary diagnostic tests could harm our business, possibly materially.
−Removed: In addition, in connection with our siRNA programs, we currently collaborate with and rely on Arrowhead to supply drug substance manufacturing and testing services for ongoing and future clinical trials.
−Removed: If in the future we seek to directly manage manufacturing of any clinical and commercial products and product candidates in our siRNA pipeline, such change may divert attention from management and could shift resources from our commercial manufacturing for our other drug products and product candidates.
Lastly, we may enter into long-term manufacturing agreements that contain exclusivity provisions and /or substantial termination penalties;
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events may continue to cause volatility in demand for ELEVIDYS, which could result in our inability to accurately forecast commercial demand for ELEVIDYS.
−Removed: In addition, if our third-party manufacturers and testing sites are unable to satisfy requirements related to the manufacturing of ELEVIDYS, our ability to meet commercial demand may be adversely impacted, which could result in the loss of potential revenues, adversely affect our ability to gain market acceptance of ELEVIDYS, or otherwise adversely affect our business, financial condition and prospects.
−Removed: Further, the physical and chemical properties of biologics such as ours generally cannot be fully characterized.
−Removed: As a result, assays of the finished product may not be sufficient to ensure that the product will perform in the intended manner.
−Removed: Accordingly, we employ multiple steps to control our manufacturing process to assure that the process works and the product candidate is made strictly and consistently in compliance with the process.
−Removed: We may encounter problems hiring and retaining the experienced scientific, quality control and manufacturing personnel needed to operate our manufacturing process, which could result in delays in our production or difficulties in maintaining compliance with applicable regulatory requirements.
−Removed: Problems with the manufacturing process, even minor deviations from the normal process, could result in delays in product release, product defects or manufacturing failures that result in lot failures, product recalls, product liability claims or insufficient inventory.
−Removed: We may encounter problems achieving adequate quantities and quality of clinical and/or commercial-grade materials that meet FDA, EMA or other applicable foreign standards or specifications with consistent and acceptable production yields and costs.
−Removed: We also face risk of damage during shipping and storage of the APIs or finished drug product.
−Removed: Lot failures or product recalls could cause us to delay clinical trials or product launches, or may result in an inability to fulfill demand for commercial supply of our products, which could be costly to us and otherwise harm our business, financial condition, results of operations and prospects.
−Removed: In addition, the FDA, the EMA and other foreign regulatory authorities may require us to submit samples of any approved product together with the protocols showing the results of applicable tests at any time.
−Removed: Under some circumstances, the FDA, the EMA or other foreign regulatory authorities may require that we not distribute a lot until the competent authority authorizes its release.
−Removed: As our product candidates advance to later stage clinical trials, it is customary that various CMC aspects of the development program, such as manufacturing, formulation and other processes, and route of administration, may be altered to optimize the candidates and processes for scale-up necessary for later stage clinical trials and potential approval and commercialization.
−Removed: These changes may not produce the intended optimization, including production of drug substance and drug product of a quality and in a quantity sufficient for clinical stage development or for commercialization, which may cause delays in the initiation or completion of clinical trials and greater costs.
−Removed: We may also need to conduct additional studies to demonstrate comparability between newly manufactured drug substance and/or drug product for commercialization relative to previously manufactured drug substance and/or drug product for clinical trials.
−Removed: Demonstrating comparability may require us to incur additional costs or delay initiation or completion of clinical trials and, if unsuccessful, could require us to complete additional pre-clinical studies or clinical trials.
−Removed: Finally, with respect to ELEVIDYS and our gene therapy programs, the current capacity to produce and test our viral vectors or gene therapy product candidates at commercial levels is limited, and the availability of sufficient GMP compliant capacity may result in delays in our development plans or increased capital expenditures, and the development and sales of any gene therapy products, if approved, may be materially harmed.
−Removed: The third parties we use in the manufacturing process for our products and product candidates may fail to comply with cGMP regulations.
−Removed: Our contract manufacturers are required to produce and test our materials, APIs and drug products under cGMP.
+Added: In addition, we announced in November 2025 topline results from our ESSENCE trial, a confirmatory trial intended to verify the clinical benefits of VYONDYS 53 and AMONDYS 45.
+Added: We submitted sNDAs related to these products in April 2026, which the FDA accepted for filing in June 2026.
+Added: Any future regulatory decision or action related to VYONDYS 53 or AMONDYS 45 could impact our previously-forecasted demand for these products, which may increase the risk of products and portions of our products’ supply expiring before sale or having excess materials on hand.
+Added: Changes in demand also may lead to changes in our commercial relationships, including pricing, that are difficult to predict and could have a negative impact on our business.
+Added: If our third-party manufacturers and testing sites are unable to satisfy requirements related to the manufacturing of our products and product candidates, our ability to meet commercial demand may be adversely impacted, which could result in the loss of potential revenues, adversely affect our ability to gain market acceptance of our products or product candidates, or otherwise adversely affect our business, financial condition and prospects.
+Added: We or the third parties we use in the manufacturing process for our products and product candidates may fail to comply with cGMP regulations.
+Added: Our contract partners are required to produce and test our materials, APIs and drug products under cGMP.
+Added: We do not have direct operational control over a third-party's compliance with regulations and requirements.
We and our contract partners are subject to periodic inspections by the FDA, EMA and corresponding state and foreign authorities to ensure strict compliance with cGMP and other applicable government regulations.
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In complying with cGMP, we are obligated to expend time, money and effort in production, record keeping and quality control to seek to assure that the product meets applicable specifications and other requirements.
−Removed: We do not have direct operational control over a third-party’s compliance with regulations and requirements.
−Removed: In addition, changes in cGMP could negatively impact the ability of our contract manufacturers to complete the manufacturing process of our products and product candidates in a compliant manner on the schedule we require for commercial and clinical trial use, respectively.
−Removed: Failure by our contract manufacturing and testing partners to achieve, maintain compliance with, or adhere to applicable cGMP and other applicable government regulations, including failure to detect or control anticipated or unanticipated errors, or our contract partners experiencing problems, may result in significant negative consequences, including product seizures or recalls, postponement
−Removed: or cancellation of clinical trials, clinical holds, loss or delay of product approvals, patient injury or death, fines and sanctions, loss of revenue, termination of the development of a product candidate, reputational damage, shipment delays, inventory shortages, inventory write-offs and other product-related charges and increased manufacturing costs.
+Added: Further, changes in cGMP could negatively impact the ability of us or our contract partners to complete the manufacturing and testing processes of our products and product candidates in a compliant manner on the schedule we require for commercial and clinical trial use, respectively.
+Added: Failure by us or our contract manufacturing and testing partners to achieve, maintain compliance with, or adhere to applicable cGMP and other applicable government regulations, including failure to detect or control anticipated or unanticipated errors, or our contract partners experiencing problems, may result in significant negative consequences, including product seizures or recalls, postponement or cancellation of clinical trials, clinical holds, loss or delay of product approvals, patient injury or death, fines and sanctions, loss of revenue, termination of the development of a product candidate, reputational damage, shipment delays, inventory shortages, inventory write-offs and other product-related charges and increased manufacturing costs.
If we experience any of these consequences, the success of our commercialization of our products and/or our development efforts for our product candidates could be significantly delayed, fail or otherwise be negatively impacted.
+Added: Finally, with respect to ELEVIDYS and our gene therapy programs in particular, the current capacity to produce and test our viral vectors or gene therapy product candidates at commercial levels is limited, and the availability of sufficient GMP compliant capacity may result in delays in our development plans or increased capital expenditures, and the development and sales of any gene therapy products, if approved, may be materially harmed.
+Added: We also rely on a third party to develop, manufacture, obtain and maintain regulatory approval for necessary diagnostic tests for ELEVIDYS.
+Added: Any delay or failure by us or our collaborators to develop, obtain or maintain regulatory approval of the necessary diagnostic tests could harm our business, possibly materially.
We may not be able to successfully optimize manufacturing of our product candidates in sufficient quality and quantity or within targeted timelines, or be able to secure ownership of intellectual property rights developed in this process, which could negatively impact the commercial success of our products and/or the development of our product candidates.
−Removed: We have historically focused on optimizing manufacturing, including for our product candidates, gene therapy and other programs.
−Removed: We may not be able to successfully increase manufacturing capacity for the production of materials, APIs and drug products, whether in collaboration with third party manufacturers or on our own, in a manner that is safe, compliant with cGMP conditions or other applicable legal or regulatory requirements, in a cost-effective manner, in a time frame required to meet our timeline for commercialization, clinical trials and other business plans, or at all.
+Added: We have historically focused on optimizing manufacturing, including for our product candidates, gene therapy, PMOs, and other programs.
+Added: For example, as our product candidates advance to later clinical stages, we have sought and may seek in the future to alter manufacturing and/or other product components and processes to optimize the products and product candidates for the scale-up necessary for later stage clinical trials, potential approval, and commercialization.
+Added: We may not be able to successfully increase manufacturing capacity or optimize manufacturing processes for the production of materials, APIs and drug products, whether in collaboration with third party manufacturers or on our own, in a manner that is safe, compliant with cGMP conditions or other applicable legal or regulatory requirements, in a cost-effective manner, in a time frame required to meet our timeline for commercialization, clinical trials and other business plans, or at all.
Challenges complying with cGMP requirements and other quality issues arise during efforts to increase or optimize manufacturing capacity and scale up production.
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Failure to successfully validate, or maintain validation of, our manufacturing processes and analytical methods or demonstrate adequate purity, stability or comparability of our products or product candidates in a timely or cost-effective manner, or at all, may undermine our commercial efforts, including by negatively impacting the commercial availability of our products and the continued development and/or regulatory approval of our product candidates, which could significantly harm our business.
−Removed: During our work with our third-party manufacturers to increase and/or optimize manufacturing capacity, they may make proprietary improvements in the manufacturing processes for our products or product candidates.
+Added: During our work with our third-party manufacturers to increase and/or optimize manufacturing capacity and processes, they may make proprietary improvements in the manufacturing or testing processes for our products or product candidates.
We may not own or be able to secure ownership of such improvements or may have to share the intellectual property rights to those improvements.
Additionally, we may need additional processes, technologies and validation studies, which could be costly and which we may not be able to develop or acquire from third parties.
−Removed: Failure to secure the intellectual property rights required for the manufacturing process needed for large-scale clinical trials or the continued development of our product candidates could cause significant delays in our business plans or otherwise negatively impact the continued development of our product candidates.
+Added: Failure to secure the intellectual property rights required for the manufacturing and testing processes needed for large-scale clinical trials or the continued development of our product candidates could cause significant delays in our business plans or otherwise negatively impact the continued development of our product candidates.
+Added: Deviations from manufacturing, testing, packaging and distribution processes and procedures could result in manufacturing delays or delays in product release, which may harm our business.
+Added: Sarepta maintains strict adherence to applicable regulatory guidance and industry standards.
+Added: We employ multiple steps to control our manufacturing and testing processes to ensure product quality, safety and compliance throughout drug development and commercial manufacturing.
+Added: Several factors could result in delays in and expenses relating to this manufacturing control process.
+Added: We or our third-party partners may encounter problems hiring and retaining the experienced scientific, quality control and manufacturing personnel needed to operate our manufacturing process, which could result in delays in our production or difficulties in maintaining compliance with applicable regulatory requirements.
+Added: Problems with the manufacturing or testing processes, even minor deviations from the normal process, could result in delays in product release, product defects or manufacturing failures that result in lot failures, product recalls, product liability claims or insufficient inventory.
+Added: We may encounter problems achieving adequate quantities and quality of clinical and/or commercial grade materials that meet FDA, EMA or other applicable foreign standards or specifications with consistent and acceptable production yields and costs, which may and has resulted in write-offs of batches of our products not meeting our quality specifications.
+Added: We also face risk of damage during shipping and storage of the APIs or finished drug product.
+Added: Lot failures or product recalls could cause us to delay clinical trials or product launches or may result in an inability to fulfill demand for commercial supply of our products, which could be costly to us and otherwise harm our business, financial condition, results of operations and prospects.
+Added: In addition, the FDA, EMA and other foreign regulatory authorities may require us to submit samples of any approved product together with the protocols showing the results of applicable tests at any time.
+Added: Under some circumstances, the FDA, the EMA or other foreign regulatory authorities may require that a product lot not be distributed until the applicable regulatory authority has authorized its release, or may prohibit its distribution altogether.
Risks Related to our Intellectual Property
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The patent positions of pharmaceutical, biotechnology and other life sciences companies can be highly uncertain and involve complex legal and factual questions for which important legal principles remain unresolved.
−Removed: This uncertainty is heightened for our PMO- based products and product candidates, our gene therapy-based products and product candidates, and our siRNA product candidates for which there has not been a significant number of patent litigations involving such technologies.
+Added: This uncertainty is heightened for our
+Added: PMO- based products and product candidates, our gene therapy-based products and product candidates, and our siRNA product candidates for which there has not been a significant number of patent litigations involving such technologies.
Congress periodically considers changes to patent law, and that such changes could have adverse effects.
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Accordingly, even if we or our licensors are able to obtain patents, the patents might be substantially narrower than anticipated.
−Removed: there is no assurance as to the degree and range of protections any of our patents, if issued, may afford us or whether patents will be issued.
+Added: Thus, there is no assurance as to the degree and range of protections any of our patents, if issued, may afford us or whether patents will be issued.
Patents which may be issued to us may be subjected to further governmental review that may ultimately result in the reduction of their scope of protection or term of patent, and pending patent applications may have their requested breadth of protection significantly limited before being issued, if issued at all.
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Litigation, interferences, oppositions, inter partes reviews, administrative challenges or other similar types of proceedings are unpredictable and may be protracted, expensive and distracting to management.
−Removed: The outcomes of such proceedings could adversely affect the validity, enforceability, and scope of our patents or other proprietary rights, hinder our ability to manufacture and market our products, require us to seek a license for the infringed products or technology or result in the assessment of significant monetary damages against us that may exceed amounts, if any, accrued in our financial statements.
+Added: The outcomes of such proceedings could adversely affect the validity,
+Added: enforceability, and scope of our patents or other proprietary rights, hinder our ability to manufacture and market our products, require us to seek a license for the infringed products or technology or result in the assessment of significant monetary damages against us that may exceed amounts, if any, accrued in our financial statements.
An adverse determination in a judicial or administrative proceeding or a failure to obtain necessary licenses could prevent us from developing, manufacturing or selling our products.
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Department of Justice (“DOJ”) issued a white collar enforcement plan identifying enforcement priorities for prosecuting corporate and white collar crime which includes health care fraud.
−Removed: Similarly, the DOJ-HHS False Claims Act Working Group enforcement priorities include drug pricing and kickbacks related to drugs paid for by federal healthcare programs.
+Added: Similarly, the DOJ-HHS False Claims Act Working
+Added: Group enforcement priorities include drug pricing and kickbacks related to drugs paid for by federal healthcare programs.
+Added: More broadly, in early 2026, the current presidential administration created a Task Force to Eliminate Fraud to coordinate and accelerate a comprehensive "whole-of-government" national strategy to stop fraud, waste, and abuse within federal benefit programs, and a new DOJ division was established for national fraud enforcement, focusing on fraud targeting federal government programs, federally funded benefits, businesses, nonprofits, and private citizens nationwide.
+Added: In June of 2026, the DOJ announced the results of the National Health Care Fraud Takedown, which included charges against 455 defendants for alleged participation in health care fraud and opioid abuse schemes.
+Added: If we were ever subject to such an investigation, we may face substantial penalties and our business, operations and financial condition could be adversely affected.
We have implemented a compliance program, which is based on industry best practices and is designed to ensure that our activities comply with all applicable laws, regulations and industry standards.
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The workforce reduction represented approximately 36% of our workforce at the time it was implemented.
−Removed: We may not realize, in full or in part, the anticipated benefits, savings and improvements in our cost structure from these efforts due to
−Removed: unforeseen difficulties, delays or unexpected costs.
+Added: We may not realize, in full or in part, the anticipated benefits, savings and improvements in our cost structure from these efforts due to unforeseen difficulties, delays or unexpected costs.
If we are unable to realize the potential cost savings from the strategic restructuring plan, our business strategy, operating results and financial condition would be adversely affected.
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All of these evolving compliance and operational requirements impose significant costs that are likely to increase over time, may require us to modify our data processing practices and policies, divert resources from other initiatives and projects and could restrict the way services involving data are offered, all of which may adversely affect our results of operations.
−Removed: Certain state laws may be more stringent or broader in scope, or offer greater individual rights, than federal or other state laws, and such laws may differ from each other, which may complicate compliance efforts.
+Added: Certain state laws may be more stringent or broader in scope, or offer greater individual rights, than federal or other state laws, and such laws may differ from
+Added: each other, which may complicate compliance efforts.
State laws are changing rapidly and there is ongoing discussion in Congress of a new federal data protection and privacy law to which we may be subject.
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We participate in the Medicaid Drug Rebate Program, the PHS 340B drug pricing program, the U.S.
−Removed: Department of Veterans Affairs, Federal Supply Schedule pricing program, and the Tricare Retail Pharmacy program, and have obligations to report the
−Removed: average sales price for certain drug products to the Medicare program.
+Added: Department of Veterans Affairs, Federal Supply Schedule pricing program, and the Tricare Retail Pharmacy program, and have obligations to report the average sales price for certain drug products to the Medicare program.
Compliance is challenging.
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Requirements are subject to challenge and change.
−Removed: For instance, the PHS 340B drug pricing program continues to be subject to legal and regulatory activity, including litigation, at the federal and state levels, and any related developments could alert the scope of the program and our obligation to offer discounts.
+Added: For instance, the PHS 340B drug pricing program continues to be subject to legal and regulatory activity, including litigation, at the federal and state levels, and any related developments could alter the scope of the program and our obligation to offer discounts.
Continued expansion of the PHS 340B drug pricing program and growth of entities claiming entitlement to 340B pricing, including in ways that may be inconsistent with the statutory scheme, could impact our revenue.
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Recent drug pricing and payment reforms initiatives under the current presidential administration such as the call for “most favored nation pricing” for products covered under government programs, if implemented, could affect our obligations under government pricing and price reporting programs.
−Removed: See “ Risks Related to Our Business—Healthcare policy reform and other governmental and private payor initiatives may have an adverse effect upon, and could prevent commercial success of our products and product candidates .” Several states have also passed or are considering legislation that requires or purports to require companies to report pricing information, including proprietary pricing information.
+Added: See “ Risks Related to Our Business—Healthcare policy reform and other governmental and private payor initiatives may have an adverse effect upon, and could prevent commercial success of our products
+Added: and product candidates .” Several states have also passed or are considering legislation that requires or purports to require companies to report pricing information, including proprietary pricing information.
Such reporting requirements are not always clearly defined and failure to appropriately disclose in accordance with these requirements may lead to the imposition of penalties.
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and abroad, report financial information or data accurately or disclose unauthorized activities to us.
−Removed: We adopted a code of conduct applicable to all of our employees, but it is not always possible to identify and deter employee misconduct, and the precautions we take to detect and prevent this activity may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to comply with these laws or
+Added: We adopted a code of conduct applicable to all of our employees, but it is not always possible to identify and deter employee misconduct, and the precautions we take to detect and prevent this activity may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to comply with these laws or regulations.
If any such actions are instituted against us, and we are not successful in defending ourselves or asserting our rights, those actions could have a significant impact on our business, including the imposition of significant fines or other sanctions.
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Over the last few years, we have had several executive management changes, including the departure of Dallan Murray, Chief Customer Officer in July 2025 and Bilal Arif, Chief Technical Operations Officer in August 2025.
−Removed: Additionally, Doug Ingram, our Chief Executive Officer announced in February 2026 his intention to retire by the end of 2026 or upon the earlier appointment of his successor.
+Added: Additionally, Doug Ingram, our former Chief Executive Officer, retired from the Company as of July 28, 2026, and Michael Severino, M.D., was appointed Chief Executive Officer.
Leadership transitions can be inherently difficult to manage and may cause uncertainty or a disruption to our business or may increase the likelihood of turnover in other key officers and employees.
Additionally, our restructuring and reduction in force in July 2025 has resulted in an increase in the turnover of employees.
−Removed: The restructuring and reduction in force could also lead to unforeseen disruptions to our business and may continue to impact the likelihood of turnover of additional employees, key employees or officers.
+Added: The restructuring and reduction in force could also lead to unforeseen disruptions to our business and may
+Added: continue to impact the likelihood of turnover of additional employees, key employees or officers.
If we lose the services of one or more of our senior management or key employees, or if one or more of them decides to join a competitor or otherwise to compete with us, our business could be harmed.
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We have previously incurred operating losses and we may not maintain profitability.
−Removed: We generated operating income of $358.4 million for the three months ended March 31, 2026.
−Removed: Our accumulated deficit was $4.6 billion as of March 31, 2026.
+Added: We generated operating income of $371.7 million for the six months ended June 30, 2026.
+Added: Our accumulated deficit was $4.6 billion as of June 30, 2026.
Although we currently have four commercially approved products in the U.S., we believe that it will take us some time to attain positive cash flow from operations.
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Our obligations under the Credit Agreement are secured by substantially all of our assets and the assets of certain wholly owned material subsidiaries, subject to certain customary exceptions and exclusions.
−Removed: The security interest granted over our assets could limit our ability to obtain additional debt financing.
+Added: The security interest granted over our assets
+Added: could limit our ability to obtain additional debt financing.
In addition, the Credit Agreement contains financial covenants that are tested on the last day of each of the Company’s fiscal quarters.
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We cannot assure you that the measures we have taken to date, and actions we may take in the future, will prevent or avoid potential material weaknesses in our internal control over financial reporting in the future.
−Removed: Any failure to maintain internal control over financial reporting, including as a result of impacts to our financial reporting team in light of our July 2025 restructuring, could severely inhibit our ability to accurately report our financial condition or results of operations and may result in a restatement of our financial statements for prior periods.
+Added: Any failure to maintain internal control over financial reporting, including as a result of impacts to our financial reporting team in light of the Restructuring, could severely inhibit our ability to accurately report our financial condition or results of operations and may result in a restatement of our financial statements for prior periods.
If we are unable to conclude that our internal control over financial reporting are effective, or if management or our independent registered public accounting firm determines we have a material weakness or significant deficiency in our internal control over financial reporting, we could lose investor confidence in the accuracy and completeness of our financial reports, the market price of our common stock could decline, and we could be subject to sanctions or investigations by the SEC or other regulatory authorities.
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The preparation of these consolidated financial statements requires us to make estimates and judgments that affect the reported amounts of our assets, liabilities, revenues and expenses, the amounts of charges accrued by us and related disclosure of contingent assets and liabilities.
−Removed: Such estimates and judgments include revenue recognition, inventory, valuation of stock-based awards, research and development expenses and income tax.
+Added: Such estimates and judgments include revenue recognition, inventory, valuation of stock-based awards, research and development expenses, income tax and the impacts of litigation, including expected settlement arrangements.
We base our estimates on historical experience, facts and circumstances known to us and on various other assumptions that we believe to be reasonable under the circumstances.
−Removed: We cannot provide assurances, however, that our estimates, or the assumptions underlying them, will not change over time or otherwise prove inaccurate.
+Added: We cannot provide assurances, however, that our
+Added: estimates, or the assumptions underlying them, will not change over time or otherwise prove inaccurate.
If this is the case, we may be required to restate our consolidated financial statements or condensed consolidated financial statements, which could, in turn, subject us to securities class action litigation.
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The Company is currently facing a securities class action litigation filed on June 26, 2025.
−Removed: In addition, related derivative lawsuits have been filed in 2025.
+Added: In addition, related derivative lawsuits have
+Added: been filed in 2025 and 2026.
Such litigation could result in substantial costs and a diversion of our management’s attention and resources.
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Currently, our Amended and Restated Certificate of Incorporation authorizes the issuance of up to 198.0 million shares of common stock.
−Removed: As of March 31, 2026, there were approximately 105.6 million shares of common stock outstanding and outstanding awards to purchase 12.0 million shares of common stock under various incentive stock plans.
−Removed: Additionally, as of March 31, 2026,
−Removed: there were approximately 2.1 million shares of common stock available for future issuance under our 2018 Equity Incentive Plan and approximately 2.2 million shares of common stock available for issuance under our 2024 Employment Commencement Incentive Plan.
+Added: As of June 30, 2026, there were approximately 105.6 million shares of common stock outstanding and outstanding awards to purchase 12.2 million shares of common stock under various incentive stock plans.
+Added: Additionally, as of June 30, 2026, there were approximately 6.6 million shares of common stock available for future issuance under our 2026 Equity Incentive Plan and approximately 2.0 million shares of common stock available for issuance under our 2024 Employment Commencement Incentive Plan.
We may issue additional shares to grant equity awards to our employees, officers, directors and consultants under our 2026 Equity Incentive Plan or our 2024 Employment Commencement Incentive Plan.
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Chambers Living Trust in a private placement.
−Removed: Additionally, on August 28, 2025, the Company completed privately negotiated exchanges of $700.0 million in aggregate principal amount of 2027 Notes for consideration consisting of (i) $602.0 million in aggregate principal amount of 2030 Notes, (ii) an aggregate of 5,851,693 shares of the Company’s Common Stock, and (iii) an aggregate of approximately $127.3 million in cash.
+Added: Additionally, on August 28, 2025, the Company completed privately negotiated exchanges of $700.0 million in aggregate principal amount of 2027 Notes for consideration consisting
+Added: of (i) $602.0 million in aggregate principal amount of 2030 Notes, (ii) an aggregate of 5,851,693 shares of the Company’s Common Stock, and (iii) an aggregate of approximately $127.3 million in cash.
On December 18, 2025, the Company completed privately negotiated exchanges of approximately $291.4 million in aggregate principal amount of 2027 Notes for consideration consisting of (i) approximately $291.4 million in aggregate principal amount of 2030 Notes and (ii) an aggregate of approximately $31.6 million in cash.
−Removed: As of March 31, 2026, the aggregate principal amount of 2027 Notes totaled approximately $158.6 million and the aggregate principal amount of 2030 Notes totaled approximately $893.4 million.
+Added: As of June 30, 2026, the aggregate principal amount of 2027 Notes totaled approximately $158.6 million and the aggregate principal amount of 2030 Notes totaled approximately $893.4 million.
Our ability to make scheduled payments of the principal of, to pay interest on, or to refinance our indebtedness, including the 2027 Notes and 2030 Notes, depends on our future performance, which is subject to many factors, including, economic, financial, competitive and other, beyond our control.
8 unchanged sentences
Our failure to repurchase the 2027 Notes or 2030 Notes upon a fundamental change when required would result in an event of default with respect to the 2027 Notes or 2030 Notes which could, in turn, constitute a default under the terms of our other indebtedness, if any.
−Removed: If the repayment of the related indebtedness
−Removed: were to be accelerated after any applicable notice or grace periods, we may not have sufficient funds to repay the indebtedness and repurchase the 2027 Notes or 2030 Notes.
+Added: If the repayment of the related indebtedness were to be accelerated after any applicable notice or grace periods, we may not have sufficient funds to repay the indebtedness and repurchase the 2027 Notes or 2030 Notes.
Capped call transactions entered into in connection with the 2027 Notes may impact the value of our common stock.
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In addition, a weak or declining economy could strain the third-parties we rely upon, including manufacturers, possibly resulting in manufacturing disruption, or cause delays in payments for our services by third-party payors or our future collaborators.
−Removed: Any of the foregoing could harm our business and we cannot anticipate all of the ways in which the current economic climate and financial market conditions could harm our business.
+Added: Any of the foregoing could harm our business
+Added: and we cannot anticipate all of the ways in which the current economic climate and financial market conditions could harm our business.
We may be subject to product liability claims and our insurance may not be adequate to cover damages.
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For example, the Foreign Corrupt Practices Act (“FCPA”) prohibits U.S.
−Removed: companies and their representatives from paying, offering to pay, promising to pay or authorizing the payment of anything of value to any foreign government official, government staff member, political party or political candidate for the purpose of obtaining or retaining business or to otherwise obtain favorable treatment or influence a person working in an official capacity.
+Added: companies and their representatives from paying, offering to pay, promising to pay or authorizing the payment of anything of value to any foreign government official, government staff member, political party or political candidate for the purpose of obtaining or retaining business or to otherwise obtain favorable treatment or influence a person working in an official
In many countries, the healthcare professionals we regularly interact with may meet the FCPA's definition of a foreign government official.
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There are risks involved in developing and using AI in our operations, including related to enhanced governmental or regulatory scrutiny and our development and use of AI may not be beneficial to our business, including the development of our product candidates or our profitability or efficiency.
−Removed: In addition, any misuse of social media or AI may result in inappropriate disclosure of sensitive information or cause reputational harm, give rise to liability, lead to the loss of trade secrets and other IP, or lead to other consequences.
+Added: In addition, any misuse of social media or AI may result in inappropriate disclosure of sensitive information or cause reputational harm, give rise to liability, lead to the loss of trade secrets and other intellectual property, or lead to other consequences.
If any of these events described above were to occur or we otherwise fail to comply with applicable regulations, we could incur liability, face overly restrictive regulatory actions or incur other harm to our business.
8 unchanged sentences
Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.