The U.S. Food and Drug Administration granted Fortress Biotech Inc.'s CUTX-101 the rare pediatric disease designation for its potential in treating a copper metabolism disorder called Menkes.
CUTX-101, or copper histidinate, is being developed by Fortress unit Cyprium Therapeutics, Inc. to treat patients with Menkes disease — a rare, genetic pediatric disorder that affects copper levels in the body. It is characterized by sparse and depigmented hair, failure to gain weight and deterioration of the nervous system.
There are no FDA-approved treatments for Menkes disease, Cyprium President and CEO Lung Yam said.
The treatment has previously received the U.S. regulator's orphan-drug and fast-track designations, all of which are meant to incentivize and expedite the development of treatments for serious or life-threatening diseases that affect a smaller segment of the population.
Cyprium plans to submit a rolling new drug application for CUTX-101 in the U.S. in the second half of 2020.