Analysts downgraded Sarepta as FDA pause on Elevidys raises regulatory uncertainty, leading to significant stock pressure and valuation cuts.

Roche is pausing shipments of Elevidys in some countries, following partner Sarepta Therapeutics’ move in the U.S., as safety concerns mount over the Duchenne muscular dystrophy gene therapy. | Roche is pausing shipments of Elevidys in some countries,

The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational gene therapy trials for limb girdle muscular dystrophy following three patient deaths potentially linked to the company's treatments.

Sarepta Therapeutics refused a FDA request to halt shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy

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.

A Cambridge-based company developing gene therapies for rare diseases is laying off more than a third of its workforce.

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.