Fortress Biotech’s partner Cyprium Therapeutics’ has received Rare Pediatric Disease Designation from the FDA for its Menkes treatment.
Fortress Biotech (NASDAQ:FBIO) announced that the US Food and Drug Administration has granted Rare Pediatric Disease Designation to its partner company Cyprium Therapeutics’ CUTX-101 for the treatment of Menkes disease.
As quoted in the press release:
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.
The FDA grants Rare Pediatric Disease Designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. If Cyprium’s new drug application (“NDA”) is approved, the company may be eligible to receive a priority review voucher, which can be redeemed to obtain priority review for any subsequent marketing application and may be sold or transferred. This program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.