Sarepta Therapeutics Announces Positive Vote from U.S. FDA Advisory Committee Meeting for SRP-9001 Gene Therapy to Treat Duchenne Muscular Dystrophy
Sarepta Therapeutics announced that the FDA Cellular, Tissue and Gene Therapies Advisory Committee voted 8-6 in favor of accelerated approval for SRP-9001, a gene therapy designed to treat Duchenne muscular dystrophy (DMD). The therapy aims to deliver a gene coding for a shortened, functional form of dystrophin to muscle cells, addressing the underlying cause of DMD.
The committee's decision was based on non-clinical evidence and data from studies 101, 102, and 103, along with an integrated analysis comparing functional results to a propensity-score-weighted external control. While not binding, this vote will be considered by the FDA in its final decision, with a regulatory action date set for May 29, 2023.
SRP-9001 is currently under priority review by the FDA. Sarepta plans to commercialize the therapy in the United States upon approval, while Roche will handle distribution outside the U.S. as part of a partnership formed in December 2019.
Sarepta Therapeutics ha annunciato che il Comitato Consultivo dell'FDA per Terapie Cellulari, Tissutali e Geniche ha votato 8-6 a favore dell'approvazione accelerata per SRP-9001, una terapia genica progettata per trattare la distrofia muscolare di Duchenne (DMD). La terapia mira a fornire un gene che codifica per una forma accorciata e funzionale della distrofina alle cellule muscolari, affrontando la causa sottostante della DMD.
La decisione del comitato si è basata su prove non cliniche e dati provenienti dagli studi 101, 102 e 103, insieme a un'analisi integrata che confronta i risultati funzionali a un controllo esterno ponderato per punteggio di propensione. Anche se non vincolante, questo voto sarà preso in considerazione dall'FDA nella sua decisione finale, con una data di azione regolamentare fissata per il 29 maggio 2023.
SRP-9001 è attualmente in fase di revisione prioritaria da parte dell'FDA. Sarepta prevede di commercializzare la terapia negli Stati Uniti dopo l'approvazione, mentre Roche gestirà la distribuzione al di fuori degli Stati Uniti come parte di una partnership formata nel dicembre 2019.
Sarepta Therapeutics anunció que el Comité Asesor de Terapias Celulares, Tisulares y Génicas de la FDA votó 8-6 a favor de la aprobación acelerada para SRP-9001, una terapia génica diseñada para tratar la distrofia muscular de Duchenne (DMD). La terapia tiene como objetivo entregar un gen que codifica una forma acortada y funcional de distrofina a las células musculares, abordando la causa subyacente de la DMD.
La decisión del comité se basó en evidencia no clínica y datos de los estudios 101, 102 y 103, junto con un análisis integrado que compara los resultados funcionales con un control externo ponderado por puntaje de propensión. Aunque no es vinculante, este voto será considerado por la FDA en su decisión final, con una fecha de acción regulatoria establecida para el 29 de mayo de 2023.
SRP-9001 está actualmente bajo revisión prioritaria por parte de la FDA. Sarepta planea comercializar la terapia en los Estados Unidos tras su aprobación, mientras que Roche se encargará de la distribución fuera de los EE.UU. como parte de una asociación formada en diciembre de 2019.
사레프타 테라퓨틱스는 FDA 세포, 조직 및 유전자 치료 자문 위원회가 SRP-9001의 가속 승인을 찬성 8표, 반대 6표로 통과시켰다고 발표했습니다. 이 유전자 치료는 두센 근육 위축증(DMD) 치료를 위해 설계되었습니다. 이 요법은 근육 세포에 단축된 기능형 디스트로핀을 코딩하는 유전자를 전달하여 DMD의 근본 원인을 다루는 것을 목표로 합니다.
위원회의 결정은 비임상 증거와 연구 101, 102 및 103의 데이터, 그리고 성과 결과를 외부 대조군과 비교한 통합 분석을 바탕으로 했습니다. 비록 구속력이 없지만, 이 표결은 FDA의 최종 결정에 고려될 것이며 규제 조치 날짜는 2023년 5월 29일로 설정되어 있습니다.
SRP-9001은 현재 FDA의 우선 심사를 받고 있습니다. 사레프타는 승인이 얻어지면 미국에서 요법을 상용화할 계획이며, 로슈는 2019년 12월에 형성된 파트너십의 일환으로 미국 외 지역의 유통을 담당할 것입니다.
Sarepta Therapeutics a annoncé que le Comité consultatif de la FDA pour les thérapies cellulaires, tissulaires et géniques a voté 8 à 6 en faveur de l'approbation accélérée de SRP-9001, une thérapie génique conçue pour traiter la dystrophie musculaire de Duchenne (DMD). Cette thérapie vise à fournir un gène codant pour une forme fonctionnelle et raccourcie de dystrophine aux cellules musculaires, traitant ainsi la cause sous-jacente de la DMD.
La décision du comité était basée sur des preuves non cliniques et des données provenant des études 101, 102 et 103, ainsi qu'une analyse intégrée comparant les résultats fonctionnels à un contrôle externe pondéré par score de propension. Bien que non contraignant, ce vote sera pris en compte par la FDA dans sa décision finale, avec une date d'action réglementaire fixée au 29 mai 2023.
SRP-9001 est actuellement soumis à un examen prioritaire par la FDA. Sarepta prévoit de commercialiser la thérapie aux États-Unis après approbation, tandis que Roche prendra en charge la distribution en dehors des États-Unis dans le cadre d'un partenariat formé en décembre 2019.
Sarepta Therapeutics gab bekannt, dass der Beratungsausschuss der FDA für Zell-, Gewebe- und Gentherapien mit 8 zu 6 für die beschleunigte Zulassung von SRP-9001 gestimmt hat, einer Gentherapie, die zur Behandlung von Duchenne-Muskeldystrophie (DMD) entwickelt wurde. Die Therapie zielt darauf ab, ein Gen, das für eine verkürzte, funktionelle Form von Dystrophin kodiert, in Muskelzellen zu übertragen und somit die zugrunde liegende Ursache der DMD anzugehen.
Die Entscheidung des Ausschusses basierte auf nicht-klinkalischen Beweisen und Daten aus den Studien 101, 102 und 103 sowie einer integrierten Analyse, die die funktionalen Ergebnisse mit einer externen Kontrollgruppe mit einem Neigungswert verglich. Obwohl nicht verbindlich, wird diese Abstimmung von der FDA bei ihrer endgültigen Entscheidung berücksichtigt, mit einem Regulierungsmaßnahmedatum, das auf den 29. Mai 2023 festgelegt ist.
SRP-9001 befindet sich derzeit im beschleunigten Prüfverfahren bei der FDA. Sarepta plant, die Therapie nach der Genehmigung in den Vereinigten Staaten zu vermarkten, während Roche die Verteilung außerhalb der USA im Rahmen einer Partnerschaft, die im Dezember 2019 gegründet wurde, übernehmen wird.
- FDA Advisory Committee voted 8-6 in favor of accelerated approval for SRP-9001
- SRP-9001 is under priority review by the FDA with a regulatory action date of May 29, 2023
- Partnership with Roche for global distribution of SRP-9001 outside the United States
- The FDA Advisory Committee vote was close (8-6), indicating some uncertainty about the therapy
- FDA's final decision is still pending, and approval is not guaranteed
– Advisory committee voted 8-6 in support of accelerated approval of SRP-9001
– Regulatory action date is May 29, 2023
“Today’s advisory committee outcome is extremely important to the patient community, who are in urgent need of new therapies,” said Doug Ingram, president and chief executive officer, Sarepta. “With the May 29 action date our top priority, we will work collaboratively with the FDA to complete the review of our BLA for SRP 9001. We extend our sincere appreciation to the families, clinicians, FDA presenters and committee members who participated in today’s panel and to all those who provided input and comments both in the written record and in the open public hearing.”
SRP-9001 is intended to treat the underlying cause of Duchenne, which is characterized by mutations in the dystrophin gene that results in the lack of dystrophin protein. In the absence of dystrophin, which is required to strengthen and protect muscles, muscles become weakened and damaged. SRP-9001 is intended to deliver a gene that codes for a shortened, functional form of dystrophin to muscle cells. The committee’s positive vote is based on the evaluation of the totality of evidence including the SRP-9001 product design as well as biological and empirical data. SRP-9001 is supported by non-clinical evidence in addition to efficacy and safety data from studies 101, 102 and 103 as well as an integrated analysis across these three clinical studies comparing functional results to a propensity-score-weighted external control (EC).
The CTGTAC’s vote, while not binding, will be considered by the FDA when making its decision regarding the potential accelerated approval of SRP-9001. The Biologics License Application (BLA) for SRP-9001 is currently under priority review by the FDA with a regulatory action date of May 29, 2023.
About SRP-9001 (delandistrogene moxeparvovec)
SRP-9001 (delandistrogene moxeparvovec) is an investigational gene transfer therapy designed to address the underlying cause of DMD through the targeted production of functional components of dystrophin in muscle tissue. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a rare, fatal neuromuscular genetic disease that occurs in approximately one in every 3,500-5,000 newborn males worldwide. DMD is caused by a change or mutation in the gene that encodes instructions for dystrophin. Symptoms of DMD usually appear in infants and toddlers. Affected children may experience developmental delays such as difficulty in walking, climbing stairs or standing from a sitting position. As the disease progresses, muscle weakness in the lower limbs spreads to the arms and other areas. Most patients require full-time use of a wheelchair in their early teens, and then progressively lose the ability to independently perform activities of daily living such as using the restroom, bathing and feeding. Eventually, increasing difficulty in breathing due to respiratory muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure. The condition is universally fatal, and patients usually succumb to the disease in their twenties.
About Sarepta Therapeutics
Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visit www.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.
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Forward-Looking Statements
This press release contains “forward-looking statements.” Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believe,” “anticipate,” “plan,” “expect,” “will,” “may,” “intend,” “prepare,” “look,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements relating to our future operations, business plans, priorities, research and development programs; SRP-9001’s potential for accelerated approval; the Company’s plans to continue working with the FDA as they complete their review of the SRP-9001 BLA; the potentially transformative benefits of SRP-9001; and that the FDA is not bound by the advisory committee recommendation but takes its advice in consideration when reviewing applications.
Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: the FDA may not approve the BLA for SRP-9001 by the application PDUFA date or at all; we may not be able to comply with all FDA requests, including with respect to our SRP-9001 BLA, in a timely manner or at all; the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business, as well as the development of our product candidates and our financial and contractual obligations; our dependence on certain manufacturers to produce our products and product candidates, including any inability on our part to accurately anticipate product demand and timely secure manufacturing capacity to meet product demand, may impair the availability of product to successfully support various programs; our data for SRP-9001 may not be sufficient for obtaining regulatory approval; success in preclinical and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or with advisory committee recommendations, or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; the commencement and completion of our clinical trials and announcement of results may be delayed or prevented for a number of reasons, including, among others, denial by the regulatory agencies of permission to proceed with our clinical trials, or placement of a clinical trial on hold, challenges in identifying, recruiting, enrolling and retaining patients to participate in clinical trials and inadequate quantity or quality of supplies of a product candidate or other materials necessary to conduct clinical trials; different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials of our product candidates are positive, these data may not be sufficient to support approval by the FDA or other global regulatory authorities; we may not be able to execute on our business plans, including meeting our expected or planned regulatory milestones and timelines, research and clinical development plans, and bringing our product candidates to market, for various reasons, many of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates, and the ongoing COVID-19 pandemic; and those risks identified under the heading “Risk Factors” in our most recent Annual Report on Form 10-K for the year ended December 31, 2022 and Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.
Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law.
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iestepan@sarepta.com
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Source: Sarepta Therapeutics, Inc.
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