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.

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