Fortress Biotech Receives Rare Pediatric Disease Designation for Menkes Disease Therapy

Rare Daily Staff

The U.S. Food and Drug Administration has granted Fortress Biotech subsidiary Cyprium Therapeutics Rare Pediatric Disease designation for copper histidinate, or CUTX-101, for the treatment of Menkes diseases.

Menkes disease is a rare X-linked recessive pediatric disease caused by genetic mutations of the copper transporter ATP7A, affecting approximately one in 100,000 newborns per year. Menkes patients have low levels of copper in their blood and brain, as well as abnormal levels of certain neurochemicals. Menkes patients have sparse and 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.

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, 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.

“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,” said Lung Yam, president and CEO of Cyprium.

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes CUTX-101 eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Swedish Orphan Biovitrum sold its Priority Review voucher to AstraZeneca for $95 million.

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