Sarepta Therapeutics' Gene Therapy Elevidys Under Priority Review by FDA for Traditional Approval

Sarepta Therapeutics Inc. recently announced that the U.S. Food and Drug Administration (FDA) has accepted and filed the company's effectiveness supplement to the Biologics License Application (BLA) for their gene therapy, ELEVIDYS (delandistrogene moxeparvovec rokl). The primary goals of this supplement are to broaden the labeled indication for ELEVIDYS and to convert its accelerated approval to a traditional approval. The FDA has granted the supplement a Priority Review, with a review goal date set for June 21, 2024.

What is ELEVIDYS?

ELEVIDYS is a single dose adeno-associated virus (AAV) based gene transfer therapy designed for intravenous infusion. Its purpose is to address the underlying genetic cause of Duchenne muscular dystrophy, a muscle-wasting disorder. The therapy has undergone evaluation in four clinical studies and has met all statutory standards for safety and effectiveness. However, it's important to note that the therapy is contraindicated in patients with certain deletions in the DMD gene. The warnings and precautions include acute serious liver injury, immune-mediated myositis, myocarditis, and pre-existing immunity against AAVrh74.

What's Next for ELEVIDYS?

The FDA has acknowledged the efficacy supplement to the Biologics License Application for ELEVIDYS, which aims to expand the approved indication of ELEVIDYS by eliminating age and ambulation restrictions. This move is a significant step in converting the approval from accelerated to traditional. The FDA has granted the supplement a Priority Review and has confirmed that they will not hold an advisory committee meeting to discuss the supplement.

Sarepta's Collaboration with Roche

Sarepta Therapeutics is currently in collaboration with Roche to transform the future for those affected by Duchenne muscular dystrophy. This partnership aims to enable those living with the disease to maintain and protect their muscle function. ELEVIDYS, being a single-dose gene transfer therapy, addresses the genetic cause of Duchenne muscular dystrophy, thereby potentially revolutionizing the treatment approach for this disorder.

The Financial Outlook of Sarepta Therapeutics

Despite the promising developments in the medical front, analysts do not anticipate Sarepta to be profitable this year. The company's market capitalization stands at 11.68 billion USD, with a revenue growth of 26.13% over the last twelve months as of Q3 2023. The quarterly revenue growth has also marked an impressive 44.1% in Q3 2023. However, the price to earnings (P/E) ratio is currently at -15.87, with an adjusted P/E ratio of -25.99. The gross profit margins stand at 7.09%, and the price to book multiple is at 15.28.

With the FDA's priority review and the potential expansion of ELEVIDYS' indications, Sarepta Therapeutics continues to make strides in the biopharmaceutical industry while striving to transform the future for the Duchenne community.