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.