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ARTICLE | Regulation

Makary: New ultrarare path will provide approval based on ‘plausible mechanism’

FDA commissioner says pathway will offer ‘conditional’ approval in very rare diseases

April 18, 2025 8:25 PM UTC

FDA Commissioner Marty Makary has announced his intention to create a new pathway for drugs that treat ultrarare conditions.

“We’re going to be rolling out a new pathway for drugs, which is a pathway based on a plausible mechanism,” Makary said in an interview Thursday with The Megyn Kelly Show. ...

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