Aduhelm backlash threatens to reverse progress in FDA’s reviews of rare and ultra-rare disease drugs

Stat News

14 September 2021 - The FDA’s approval of Aduhelm to treat Alzheimer’s disease has unleashed criticism about the decision and calls for reform within the agency. 

As advocates for people living with Barth syndrome — an ultra-rare, life-threatening genetic condition affecting just 130 children and young adults in the U.S. — we fear the pushback will discourage the FDA from consistently exercising appropriate flexibility in its approval standards and stall progress being made in employing new regulatory strategies to improve access to safe and effective therapies.

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Michael Wonder

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Michael Wonder

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Medicine , US , Orphan drug , Regulation