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Keeping Track: Orphan Drugs Aplenty At US FDA, From Pyrukynd Approval To Adagrasib Standard Review

 
• By Bridget Silverman

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

Keeping Track Feature image

The US Food and Drug Administration’s approval of Agios Pharmaceuticals, Inc.’s Pyrukynd (mitapivat) capped a strong week for rare diseases at the agency.

Three of the six novel agents approved in 2022 so far by the Center for Drug Evaluation and Research have been for orphan drugs, including both February novel approvals – Pyrukund for pyruvate kinase deficiency on 17 February and

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