Sarepta Therapeutics' Therapy Becomes First To Score FDA Nod For Duchenne Muscular Dystrophy Gene Therapy

The FDA has granted accelerated approval to closely watched Sarepta Therapeutics Inc's SRPT gene therapy, Elevidys, for Duchenne muscular dystrophy (DMD) in ambulatory pediatric patients aged 4 through 5 years with a confirmed mutation in the DMD gene.

The approval is based on the observed expression of Elevidys micro-dystrophin protein in patients treated with the gene therapy. 

Continued approval for this indication will depend on further verification and description of clinical benefit in confirmatory trials.

Elevidys targets the root genetic cause of Duchenne, which is mutations in the dystrophin gene resulting in the absence of dystrophin protein. The therapy delivers a gene that codes for a shortened form of dystrophin to muscle cells.

Elevidys has shown an increase in micro-dystrophin protein expression in skeletal muscle.

Before the adcomm meeting, the FDA staff did raise some safety issues saying 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. 

Sarepta Therapeutics has committed to conducting a confirmatory trial called EMBARK, a global Phase 3 trial fully enrolled and expected to provide top-line results in late 2023.

Sarepta is preparing to launch Elevidys and plans to submit a BLA (Biologics License Application) supplement to expand the approved label based on the results of the confirmatory trial.

Price Action: SRPT shares are down 1.26% at $125.27 on the last check Thursday.