Sarepta Therapeutics Announces Positive Vote from U.S. FDA Advisory Committee Meeting for SRP-9001 Gene Therapy to Treat Duchenne Muscular Dystrophy

CAMBRIDGE, Mass. ~ Today, the U.S. Food and Drug Administration (FDA) Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) voted 8 to 6 in support of accelerated approval of SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulatory patients with Duchenne muscular dystrophy with a confirmed mutation in the DMD gene. The vote was a major milestone for Sarepta Therapeutics, Inc., a leader in precision genetic medicine for rare diseases.

Doug Ingram, president and chief executive officer of Sarepta, expressed his 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. He said: "Today's advisory committee outcome is extremely important to the patient community, who are in urgent need of new therapies. 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."

SRP-9001 is intended to treat Duchenne muscular dystrophy (DMD), which is characterized by mutations in the dystrophin gene that results in a lack of dystrophin protein. In its absence, 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 was based on an evaluation of evidence including product design as well as biological and empirical data from studies 101, 102 and 103 as well as an integrated analysis across these three clinical studies comparing functional results to an external control (EC).

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The CTGTAC's vote will be considered by the FDA when making its decision regarding 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 29th 2023.

SRP-9001 is supported by Roche who partnered with Sarepta last December 2019 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.

Duchenne muscular dystrophy affects approximately one in every 3,500-5,000 newborn males worldwide. Symptoms usually appear during infancy or toddlerhood such as difficulty walking or standing from a sitting position; eventually leading up to full time use of wheelchairs during early teens due to muscle weakness spreading throughout arms and other areas; difficulty breathing due to respiratory muscle dysfunction; cardiac dysfunction leading up heart failure; ultimately resulting in death during their twenties due to lack of treatment options available until now.

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Sarepta Therapeutics holds leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), currently having more than 40 programs in various stages of development driven by their multi-platform Precision Genetic Medicine Engine which includes gene therapy, RNA and gene editing technologies among others. They are responsible for global development and manufacturing for SRP-9001 while planning on commercializing it upon receiving FDA approval within US borders while Roche takes care outside US borders through their partnership agreement last December 2019 .

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