Eye­ing ac­cel­er­at­ed ap­proval for Duchenne gene ther­a­py, Sarep­ta hopes to bring in $1B in bid for prof­itabil­i­ty

On Mon­day, Al­ny­lam an­nounced that it was look­ing to raise $900 mil­lion on the back of a pos­i­tive read­out for its drug in AT­TR amy­loi­do­sis with car­diomy­opa­thy.

To­day, it’s Sarep­ta’s turn.

The Duchenne mus­cu­lar dy­s­tro­phy-fo­cused biotech is seek­ing $1 bil­lion ahead of a po­ten­tial ac­cel­er­at­ed ap­proval for its gene ther­a­py, which it post­ed a gen­er­al­ly pos­i­tive read­out for in Ju­ly — though it did re­port a new case of my­ocardi­tis. Fol­low­ing that read­out, Sarep­ta said it was plan­ning to sub­mit for ac­cel­er­at­ed ap­proval with the FDA, and an­a­lysts said an ad­comm was a “near cer­tain­ty.”

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER