As Sarep­ta ex­ecs talk up Duchenne con­fir­ma­to­ry tri­al re­sults, in­vestors and an­a­lysts re­main di­vid­ed on how flex­i­ble FDA will be

Any­one lis­ten­ing to Sarep­ta’s call Mon­day af­ter the biotech re­leased tri­al da­ta for its Duchenne mus­cu­lar dy­s­tro­phy gene ther­a­py would have thought the com­pa­ny hit a home run.

“The EM­BARK re­sults have met the stan­dard for sub­stan­tial ev­i­dence of ef­fec­tive­ness, con­firm­ing the mech­a­nism of ac­tion of Ele­v­idys, and show­ing that it al­ters the course of dis­ease,” CEO Doug In­gram said of the tri­al, which was de­signed to give da­ta on whether the drug’s ac­cel­er­at­ed ap­proval could be con­vert­ed in­to a full one, po­ten­tial­ly for more pa­tients.

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