FDA Vote in Favor of Accelerating Approval of Gene Therapy for Duchenne Muscular Dystrophy

The Company received a positive vote of 8 to 6 from the FDA Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) in support of the accelerated approval of its gene therapy SRP-9001 for the treatment of Duchenne muscular dystrophy.

DMD is a rare, fatal neuromuscular genetic disease that occurs in about one in every 3,500-5,000 newborn males worldwide.

“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 CEO, Sarepta.

The advisory committee has recommended the approval of Sarepta’s therapy for the treatment of DMD patients who have specific genetic mutations amenable to exon 45 skipping. These mutations are present in approximately 8% of individuals with DMD.

SRP-9001 is an antisense oligonucleotide specifically designed to skip exon 45 during the production of dystrophin, a critical protein essential for muscle function. By skipping exon 45, the gene therapy aims to restore the reading frame of the DMD gene, enabling the production of a partially functional dystrophin protein.

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The advisory committee’s decision was based on compelling data from a clinical study involving 43 DMD patients who received treatment with SRP-9001. The study demonstrated a significant increase in dystrophin production compared to a placebo, providing encouraging results.

The committee’s positive recommendation is a significant step in the regulatory process. The FDA will carefully consider the advisory committee’s input when making the final decision on the approval.

“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,” adds Doug Ingram.

Source: Sarepta Press Release

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