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Orphans Dominate New US FDA Approvals, Thanks To Rethymic, Tavneos, And Livmarli

 
• By Bridget Silverman

The latest drug development news and highlights from the Pink Sheet’s US FDA Performance Tracker.

Keeping Track Feature image

Orphan drugs account for about one-third of the 41 novel approvals granted by the FDA’s Center for Drug Evaluation and Research so far in 2021, a ratio that is replicated in the center’s three most recent approvals. ChemoCentryx, Inc.’s Tavneos and Mirum Pharmaceuticals, Inc.’s Livmarli are approved for rare diseases, while AbbVie Inc.’s Qulipta targets the sizable (and crowded) migraine space.

The Center for Biologics Evaluation and Research contributed a novel approval for Enzyvant Therapeutics, Inc.’s thymus tissue-based regenerative medicine Rethymic, making it the first treatment for the ultra-rare condition congenital athymia

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