Regulatory Affairs

Cyprium Therapeutics’ CUTX-101 gets FDA fast track designation to treat classic Menkes disease

Published 04 July 2018

Fortress Biotech announced that the US Food and Drug Administration (FDA) has granted fast track designation to Cyprium Therapeutics Copper Histidinate, also referred to as CUTX-101, for patients diagnosed with classic Menkes disease who have not demonstrated significant clinical progression.

As a subsidiary of Fortress, Cyprium is developing CUTX-101 for Menkes disease under a Cooperative Research and Development Agreement (CRADA) with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), part of the National Institutes of Health (NIH).

The CRADA was established in March 2017. Stephen G. Kaler, M.D., Senior Investigator and Head, Section on Translational Neuroscience and former NICHD Clinical Director, leads the Menkes disease research program at NICHD and serves as principal investigator of the CUTX-101 Phase 3 clinical trial.

“We are pleased that the FDA granted Fast Track Designation for Copper Histidinate based on the promising clinical data observed to date,” stated Lung S. Yam, M.D., Ph.D., President and Chief Executive Officer of Cyprium.

 “Menkes disease is a rare and fatal pediatric disease, and patients with classic Menkes disease currently have no approved therapeutic options. This designation signifies the FDA’s recognition of the significant unmet medical need that Copper Histidinate has potential to address. 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.” 

The FDA's Fast Track program facilitates the development of drugs intended to treat serious conditions and that have the potential to address unmet medical needs.

 A drug program with Fast Track status is afforded greater access to the FDA for the purpose of expediting the drug's development, review and potential approval.

 In addition, the Fast Track program allows for eligibility for Accelerated Approval and Priority Review, if relevant criteria are met, as well as for Rolling Review, which means that a drug company can submit completed sections of its New Drug Application (NDA) for review by the FDA, rather than waiting until every section of the NDA is completed before the application is submitted for review.

CUTX-101 is in development to supplement blood and brain copper levels in patients with Menkes disease and related disorders. CUTX-101 is a subcutaneous injectable formulation of Copper Histidinate manufactured under cGMP that is intended to improve tolerability due to physiological pH and bypass the oral absorption of copper, which is impaired in patients with Menkes disease. In a Phase 1/2 clinical study conducted by Dr. Kaler at NIH, early treatment of Menkes patients with CUTX-101 led to an improvement in neurodevelopmental outcomes and survival. A Phase 3 study of CUTX-101 in Menkes disease is ongoing. 

Cyprium and NICHD are also collecting natural history data on classic Menkes disease patients who were not treated with copper supplements. Data from this natural history study will serve as a historical control cohort to further demonstrate the efficacy and safety of CUTX-101.

CUTX-101 previously has been granted orphan drug designation by the U.S. Food and Drug Administration.

Menkes disease is a rare X-linked pediatric disease caused by gene mutations of copper transporter ATP7A, which affects approximately one in 100,000 newborns per year. Biochemically, Menkes patients may have low levels of copper in their blood and brain, as well as abnormal levels of catecholamines, but definitive diagnosis is typically made by sequencing the ATP7A gene.

Source: Company Press Release