CUTX-101 gains Rare Pediatric Disease designation

A coveted Rare Pediatric Disease (RPD) designation has been issued to Fortress Biotech’s (Nasdaq: FBIO) partner for its investigational Menkes disease candidate CUTX-101, with the news pushing the parent firm’s shares up more than 18% to $2.81 by close of trading Thursday.

The US Food and Drug Administration has granted RPD designation to Cyprium Therapeutics’ copper histidinate, also referred to as CUTX-101, for the treatment of Menkes disease.

Menkes disease is a rare X-linked recessive pediatric disease caused by genetic mutations of the copper transporter, ATP7A. The FDA previously granted Orphan Drug and Fast Track designations to CUTX-101 for the treatment of Menkes disease.