Rare disease designation for CUTX-101 to treat Menkes disease

March 6, 2020 0 By FM

Fortress Biotech said that the US FDA has granted Rare Paediatric Disease Designation to Cyprium Therapeutics’ copper histidinate 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.

CUTX-101 is in clinical development to supplement blood and brain copper levels in patients diagnosed with classic Menkes disease who have not demonstrated significant clinical progression.