Stealth Elamipretide NDA Tests Frontiers Of US FDA’s Flexibility In Rare Diseases
FDA wants another trial in ultra-orphan Barth syndrome, but could not identify a feasible design – so Stealth submitted the NDA anyways, based on a retrospective natural history control trial
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Keeping Track: A Lawsuit-Driven Complete Response Letter, A Refuse To File Letter, And Some Good News
The latest drug development news and highlights from the Pink Sheet’s US FDA Performance Tracker
First FDA-hosted workshop on rare family of mitochondrial diseases offers plenty of opportunities to talk challenges in R&D – and identify some solutions.
Agency specifies several approaches to augment safety assessment in a new draft guidance, which also encourages participation by patients and their advocates in the drug development process.