FDA Grants Fast Track Designation to CUTX-101 for Classic Menkes Disease

This morning, July 2, 2018, the US Food and Drug Administration (FDA) granted Cyprium Therapeutics, a subsidiary of Fortress Biotech, Inc, Fast Track Designation for its product, CUTX-101, for the treatment of patients with classic Menkes disease who have not demonstrated significant clinical progression.

CUTX-101 is a subcutaneous injectable formulation of Copper Histidinate manufactured under cGMP, according to a recent news release. It is intended to improve tolerability due to physiological pH as well as bypass the oral absorption of copper, a functionality that is impaired in patients with Menkes disease.

“Menkes disease is a rare and fatal pediatric disease, and patients with classic Menkes disease currently,” shared Lung S. Yam, MD, PhD, president and chief executive officer of Cyprium in a recent statement. “This designation signifies the FDA’s recognition of the significant unmet medical need that Copper Histidinate has potential to address.”

Under a Cooperative Research and Development Agreement (CRADA) with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)—which is a part of the National Institutes of Health (NIH)—Cyprium is developing CUTX-101 for Menkes disease. The agreement was established in March 2017.

The CUTX-101 phase 3 clinical trial is being led by Stephen G. Kaler, MD, who is the senior investigator and head of the section on Translational Neuroscience and former clinical director of the NICHD; Dr Kaler currently leads the Menkes disease research program at NICHD.

“We appreciate our ongoing discussions with the FDA and look forward to working closely with the agency and Dr Kaler as we advance the development of Copper Histidinate, which is currently being evaluated in a phase 3 study,” Cyprium’s president and chief executive officer Lung S. Yam, MD, PhD commented.

Previously, in a phase 1/2 study led by Dr Kaler at NIH, early treatment with CUTX-101 of Menkes patients displayed an improvement in neurodevelopmental outcomes and survival; the drug was granted Orphan Drug Designation by the FDA in September 2017.