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...
Deal Watch: Gemini Goes Through Second Re-Invention Via Reverse Merger With Disc Medicine
Less than two year after going public by a SPAC merger, Gemini will reverse merge with hematology-focused Disc. Bristol Myers Squibb and GentiBio will join forces on Treg development for inflammatory bowel disorders.
Four Major Clinical Trial Readouts To Watch Out For In Q2
Four prominent drug candidates are expecting topline mid- or late-stage data over the next few months. With the help of Biomedtracker's Q2 Outlook Report, we take a look at the assets that could help validate some of the companies developing them, from oncology to psychiatry.
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