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Fortress Biotech Announces Rare Pediatric Disease Designation for CUTX-101 for the Treatment of Menkes Disease
Cyprium Therapeutics, a Fortress partner company, on track to begin submitting rolling New Drug Application for CUTX-101 to the FDA in the second half of 2020
About this update from Fortress Biotech, Inc.
[{"type":"text","content":"Cyprium Therapeutics, a Fortress partner company, on track to begin submitting rolling New Drug Application for CUTX-101 to the FDA in the second half of 2020\nNEW YORK, Jan. 16, 2020 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”), an innovative biopharmaceutical company focused on identifying, in-licensing and developing high-potential marketed and development-stage drugs and drug candidates, today announced that the U.S. 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.\n 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 program 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.” About Menkes Disease and Related Copper Metabolism DisordersMenkes 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 brai...