FDA grants Rare Pediatric Disease designation to (Z)-endoxifen for treating McCune-Albright Syndrome in girls

Atossa Therapeutics received FDA Rare Pediatric Disease designation for (Z)-endoxifen to treat McCune-Albright Syndrome (MAS), a rare pediatric endocrine disorder causing early puberty and bone issues in girls. This designation may qualify Atossa for a valuable Priority Review Voucher upon drug approval, which can speed future FDA reviews or be sold. The move highlights (Z)-endoxifen's potential beyond oncology and supports Atossa's collaboration with patient groups and researchers to develop new therapies for MAS, addressing significant unmet medical needs.