Doug Ingram, Sarepta CEO (Sarepta)

FDA knocks back Sarep­ta in Duchenne gene ther­a­py race with Pfiz­er, but an­a­lysts urge cau­tion

Sarep­ta has gone all-in on gene ther­a­py over the last few years, rac­ing with Pfiz­er and Sol­id Bio­sciences to be the first to de­vel­op a ge­net­ic fix for Duchenne mus­cu­lar dy­s­tro­phy, one of the most com­mon rare dis­eases.

Sarep­ta has been com­fort­ably in the lead, col­lect­ing the first pos­i­tive re­sults and snar­ing a $1.15 bil­lion cash com­mer­cial­iza­tion deal with Roche, but this week the com­pa­ny hit a snag. Late yes­ter­day, Sarep­ta pro­vid­ed a “pro­gram up­date” for its gene ther­a­py, re­veal­ing that in a sched­uled meet­ing the FDA had raised con­cerns about the kinds of tests they would use to mea­sure po­ten­cy in the piv­otal study and com­mer­cial sup­ply for the gene ther­a­py. The com­pa­ny has as­says that might fit the cri­te­ria, they said, but “need­ed ad­di­tion­al di­a­logue” with the agency to con­firm.

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