Fortress Biotech, an innovative biopharmaceutical company focused on identifying, in-licensing and developing high-potential marketed and development-stage drugs and drug candidates, announced that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to Cyprium Therapeutics’ (Cyprium) Copper Histidinate, also referred to as CUTX-101, for the treatment of Menkes disease. Menkes disease is a rare X-linked recessive pediatric 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. The FDA grants Rare Pediatric Disease Designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. If Cyprium’s new drug application (NDA) is approved, the company may be eligible to receive a priority review voucher, which can be redeemed to obtain priority review for any subsequent marketing application and may be sold or transferred. This programme is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. “The FDA’s Rare Pediatric Disease Designation of CUTX-101 for the treatment of Menkes disease, after granting Orphan Drug and Fast Track Designations to CUTX-101 previously, highlights the significant unmet medical needs for patients with this devastating and life-threatening disease. Currently, there is no FDA-approved treatment for Menkes disease,” said Lung S. Yam, M.D., Ph.D., president and chief executive officer of Cyprium. “The entire Cyprium team is encouraged by this designation and will continue to work diligently toward submitting the NDA for CUTX-101.” Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of copper transporter ATP7A, which affects approximately one in 100,000 newborns per year. Biochemically, Menkes patients have low levels of copper in their blood and brain, as well as abnormal levels of certain neurochemicals. Definitive diagnosis is typically made by sequencing the ATP7A gene. The condition is characterized by distinctive clinical features, including sparse and depigmented hair (kinky hair), connective tissue problems, and severe neurological symptoms such as seizures, hypotonia, and failure to thrive. Mortality is high, with many patients dying before the age of three years, if untreated. Milder versions of ATP7A mutations are associated with other conditions, including Occipital Horn Syndrome and ATP7A-related Distal Motor Neuropathy. Currently, there is no FDA-approved treatment for Menkes disease and its variants. 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. CUTX-101 is a subcutaneous injectable formulation of Copper Histidinate manufactured under cGMP that is intended to improve tolerability due to physiological pH and to bypass the oral absorption of copper, which is impaired in patients with Menkes disease. In a phase 1/2 clinical trial conducted at the National Institutes of Health (NIH), early treatment of patients with Menkes disease with CUTX-101 led to an improvement in neurodevelopmental outcomes and survival. A phase 3 trial of CUTX-101 in patients with Menkes disease is ongoing at the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).