What's next for Sarep­ta? A third DMD ap­proval, an­a­lysts pre­dict

What Sarep­ta wants, Sarep­ta usu­al­ly gets.

In dra­mat­ic fash­ion on Thurs­day, the ap­proval of Vyondys 53 — to treat a sub­set of Duchenne mus­cu­lar dy­s­tro­phy (DMD) pa­tients — was un­veiled, four months af­ter the FDA’s ini­tial re­jec­tion. With two drugs now ap­proved on the ba­sis of ~1% ex­pres­sion of dy­s­trophin — the miss­ing pro­tein that caus­es DMD — Sarep­ta is in a prime po­si­tion to take its third DMD drug to the reg­u­la­tor, an­a­lysts said, pre­dict­ing health­i­er odds of suc­cess.

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