Sarepta Therapeutics Announces that the Date of the Advisory Committee Meeting for SRP-9001 will be May 12, 2023

Rhea-AI Summary

Sarepta Therapeutics (NASDAQ: SRPT) announced the date of the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee meeting for the SRP-9001 biologics license application (BLA) on May 12, 2023. This virtual meeting is critical as it precedes the regulatory action date of May 29, 2023, for the investigational gene therapy targeting Duchenne muscular dystrophy.

CEO Doug Ingram expressed anticipation in showcasing the evidence of SRP-9001’s transformative potential. The therapy aims to deliver functional dystrophin components to muscle tissue, essential for patients suffering from Duchenne muscular dystrophy. Sarepta plans to commercialize SRP-9001 in the U.S. post FDA approval and has partnered with Roche to enhance global access to this promising therapy.

Positive

• FDA Advisory Committee meeting for SRP-9001 scheduled for May 12, 2023, indicating progress towards potential approval.
• Positive statements from leadership regarding the transformative potential of SRP-9001 for Duchenne muscular dystrophy.

Negative

• Dependent on FDA approval, which carries inherent risks and uncertainties.
• If SRP-9001 data is insufficient, regulatory approval may be denied.

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the date of the U.S. Food and Drug Administration’s Cellular, Tissue and Gene Therapies Advisory Committee meeting for the SRP-9001 (delandistrogene moxeparvovec) biologics license application (BLA) is May 12, 2023. The advisory committee meeting will be hosted as a virtual meeting. SRP-9001 is Sarepta’s investigational gene therapy for the treatment of Duchenne muscular dystrophy.

“We look forward to sharing the wealth of evidence supporting the transformative potential of SRP-9001 for the treatment of Duchenne muscular dystrophy with the advisory committee on May 12, 2023,” said Doug Ingram, president and chief executive officer, Sarepta. “We would again like to thank Center for Biologics Evaluation and Research and the Office of Therapeutic Products for working swiftly to schedule the advisory committee in advance of our regulatory action date of May 29, 2023.”

About SRP-9001 (delandistrogene moxeparvovec)

SRP-9001 (delandistrogene moxeparvovec) is an investigational gene transfer therapy intended to deliver SRP-9001 to muscle tissue for the targeted production of functional components of dystrophin. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States upon receiving FDA approval. In December 2019, Roche partnered with Sarepta to combine Roche’s global reach, commercial presence and regulatory expertise with Sarepta’s gene therapy candidate for Duchenne to accelerate access to SRP-9001 for patients outside the United States.

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.

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

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; the potentially transformative benefits of SRP-9001; and expected timelines, plans and milestones, including the regulatory action date of May 29, 2023, and advisory committee meeting date of May 12, 2023.

These forward-looking statements involve risks and uncertainties, many of which are beyond our control. 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 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 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 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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20230404005793/en/

Investor:

Ian Estepan, 617-274-4052

iestepan@sarepta.com

Media:

Tracy Sorrentino, 617-301-8566

tsorrentino@sarepta.com

Source: Sarepta Therapeutics, Inc.

FAQ

What is the FDA meeting date for Sarepta's SRP-9001?

The FDA meeting for Sarepta's SRP-9001 is scheduled for May 12, 2023.

What is SRP-9001 used for?

SRP-9001 (delandistrogene moxeparvovec) is a gene therapy aimed at treating Duchenne muscular dystrophy.

When is the regulatory action date for SRP-9001?

The regulatory action date for SRP-9001 is May 29, 2023.

What are the potential risks associated with SRP-9001?

Risks include potential failure to obtain FDA approval if the data is insufficient.

Who is the CEO of Sarepta Therapeutics?

Doug Ingram is the CEO of Sarepta Therapeutics.