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
You may also be interested in...
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Agency specifies several approaches to augment safety assessment in a new draft guidance, which also encourages participation by patients and their advocates in the drug development process.
The latest drug development news and highlights from the Pink Sheet’s US FDA Performance Tracker.