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FDA Rejects BioMarin's Duchenne Treatment

The FDA's decision to reject Biomarin's drug for Duchenne muscular dystrophy comes on the heels of an FDA Advisory Committee meeting in November in which experts expressed concern about its efficacy and side effects. An Advisory Committee meeting for "eteplirsen," a similar antisense oligo-based drug for Duchenne developed by Sarepta will take place on January 22nd.

Read more about that meeting here.

Read the Reuters article on the Kendrisa rejection here.






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