rare pediatric disease
Satellos Bioscience Earns Third FDA Designation for Duchenne Drug SAT-3247
The FDA approved 44 novel therapies in the first half of 2026, including 26 in the final six months alone. BioPharma Dive described it as an acceleration, driven partly by a White House-directed overhaul aimed at restoring more traditional leadership to the agency. The pace matters for small companies whose entire business model rests on…
Breakthrough: Atossa’s (Z)-Endoxifen Gains FDA Orphan Status for DMD
Atossa Therapeutics (Nasdaq: ATOS) said the FDA granted Orphan Drug Designation for its drug (Z)-endoxifen in Duchenne muscular dystrophy. This follows a prior Rare Pediatric Disease designation. It’s a positive regulatory step. But it does not guarantee approval and it does not make trials any faster. CEO Steven Quay called it an important milestone. He…
