Impasse At US FDA Could Mean Stealth Abandons Barth Syndrome Treatment
In an interview with the Pink Sheet, Stealth’s CEO says she is not confident the company could design a clinical trial in the rare disease that would meet the FDA’s expectations.
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The group is intended to help rare disease drug developers answer all the necessary questions about surrogate endpoints and markers before the application review cycle.
Supplemental Filings: FDA Can't 'Plug' All Shortage Holes, New Jobs For Billy Dunn, Lisa Parks & Heidi Rebello
From infant formula to drugs, Califf can't get away from shortage work. Plus, the revolving door brings new jobs for some top pharma folks.
Pink Sheet Drug Review Profile looks at how the agency determined that Amylyx’s ‘conceptual basis’ for use of the drug’s two active ingredients to treat ALS was enough to satisfy the combination drug rule; the sponsor also was allowed to submit certain animal studies and drug-drug interaction data during the course of the NDA review.