Posted by Michael Wonder on 17 Jan 2020
Fortress Biotech announces rare paediatric disease designation for CUTX-101 for the treatment of Menkes disease
16 January 2020 - Cyprium Therapeutics, a Fortress partner company, on track to begin submitting rolling new drug application for CUTX-101 to the FDA in the second half of 2020.
Fortress Biotech today announced that the U.S. FDA has granted Rare Pediatric Disease 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 paediatric 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.
