US FDA Still Mulling Over Sarepta’s Duchenne’s Gene Therapy

The US Food and Drug Administration (FDA) has yet to make a call on Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy, although it appears to be leaning towards a limited “yes”. As a result, the agency has pushed back its decision from May 29^th to June 22^nd. Deliberations will continue in the meantime, and some expect approval for a limited age range.

According to Annalee Armstrong, “The restricted age range would reflect the FDA’s concerns that Sarepta did not have enough evidence to support use in older patients. In briefing documents prepared for the advisory meeting, the FDA said: “It is challenging to conclude with reasonable certainty from the data provided by the applicant either that SRP-9001 is likely effective for younger patients, or that it is likely ineffective for older patients or those with somewhat poorer functional status.””

To read more, click here.

(Source: Fierce Biotech, May 24^th, 2023)