FDA Grants Orphan-Drug Designation To Angelman Syndrome Treatment

The U.S. Food and Drug Administration (FDA) recently granted orphan-drug designation for GTX-101, GeneTx Biotherapeutics LLC’s drug intended for the treatment of Angelman Syndrome. Angelman syndrome is a complex genetic disorder that primarily affects the nervous system.

The Orphan Drug Designation Program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis, or prevention of rare diseases/disorders that affect few than 200,000 U.S. citizens, or that affect more than 2000,000 persons but are not expected to recover the costs of developing and . . .