FDA AdComm Narrowly Supports Approval Of Sarepta's Gene Therapy For Rare Neuromuscular Disorder

On Friday, FDA's Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) voted 8 to 6 in support of accelerated approval of Sarepta Therapeutics Inc's SRPT SRP-9001 (delandistrogene moxeparvovec) for ambulatory patients with Duchenne muscular dystrophy.

The FDA is slated to make a decision on accelerated approval by May 29. The agency typically follows the advice of its expert advisers but is not obligated to do so.

"Today's advisory committee outcome is extremely important to the patient community," Sarepta's CEO Doug Ingram said in a statement.

Sarepta is hoping to gain approval through the FDA's accelerated pathway.

SRP-9001 is also being studied in EMBARK Phase 3 Study, which has recruited 126 participants with Duchenne between the ages of 4 to 7. Results from EMBARK are expected by the end of 2023. Sarepta has proposed EMBARK as the post-marketing confirmatory trial for SRP-9001.

In briefing documents, the FDA said the data does not provide unambiguous evidence that SRP-9001 is likely beneficial for ambulatory patients with Duchenne muscular dystrophy.

The FDA staff says it is challenging to conclude with reasonable certainty from the data that SRP-9001 is likely effective for younger patients or is likely ineffective for older patients or those with somewhat poorer functional status. 

Price Action: SRPT shares closed at $120.20 on Friday.