Stealth Elamipretide NDA Tests Frontiers Of US FDA’s Flexibility In Rare Diseases
Executive Summary
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...
Mitochondrial Disease Workshop Shows US FDA Commitment To First Approvals
First FDA-hosted workshop on rare family of mitochondrial diseases offers plenty of opportunities to talk challenges in R&D – and identify some solutions.
Rare Diseases: US FDA Gives More Guidance On Premarket Safety Evaluation, Historical Controls
Agency specifies several approaches to augment safety assessment in a new draft guidance, which also encourages participation by patients and their advocates in the drug development process.
Keeping Track: New Formulations Dominate In CNS; Immunocore Brings First TCR Therapy To US FDA
The latest drug development news and highlights from the Pink Sheet’s US FDA Performance Tracker.