Sarepta faces an “arduous and treacherous path” to try to get its Duchenne muscular dystrophy therapy Elevidys back onto the market

Sarepta Therapeutics faces serious FDA action after news broke of a third patient death, the FDA gets a new top drug regulator in George Tidmarsh, a handful of new drugs get turned away from the market and pharma companies continue to commit billions to reshoring manufacturing.

The Sarepta saga continues, with the FDA slapping a clinical hold across all of the company’s investigational limb-girdle muscular dystrophy (LGMD) trials, while also revoking the biopharma’s gene therapy platform technology designation.

Sarepta Therapeutics (NASDAQ:SRPT) faces an “arduous and treacherous path” to bring its gene therapy Elevidys back to market, according to a STAT report citing a senior FDA official, suggesting the treatment’s license could be revoked amid ongoing safety concerns.

Discovered and developed by Sarepta Therapeutics, Elevidys is a gene therapy that won accelerated approval in June 2023 and full approval in June 2024. Roche partnered with Sarepta in December 2019, paying $1.15 billion upfront in cash and stock for the right to launch and market Elevidys outside the U.S.

Arrowhead Pharmaceuticals said on Wednesday it expects to receive near-term milestone payments from Sarepta Therapeutics as part of their licensing agreement, despite recent setbacks at the Cambridge,

Sarepta stock took another dive Monday after the biotech company refused the FDA's request to voluntarily stop all shipments of Elevidys.

The FDA will request Sarepta Therapeutics stop all shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy, following two deaths tied to the product.