Why Star Therapeutics’ FDA win for VGA039 could reshape the von Willebrand disease treatment race.
Star Therapeutics won dual FDA designations for VGA039 in von Willebrand disease. Read what it could change, and what still needs proving.
FDA grants orphan drug designation to Atossa’s (Z)-Endoxifen for Duchenne: What’s next?
Atossa Therapeutics Inc. has received Orphan Drug Designation from the U.S. Food and Drug Administration for its investigational compound (Z)-Endoxifen in the treatment of Duchenne muscular dystrophy (DMD). The designation, disclosed on January 16, 2026, supplements the previously granted Rare Pediatric Disease Designation for the same compound and indication. The move marks a notable regulatory […]
Atossa pivots into rare disease with FDA nod for (Z)-Endoxifen in Duchenne muscular dystrophy
Atossa Therapeutics has received Rare Pediatric Disease (RPD) designation from the U.S. Food and Drug Administration for (Z)-Endoxifen in Duchenne muscular dystrophy (DMD), a strategic shift that positions the estrogen receptor modulator for possible use in pediatric neuromuscular disorders. If approved, the program could qualify for a transferable Priority Review Voucher, but the timeline and […]