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Fortress Biotech Announces Breakthrough Therapy Designation for CUTX-101, Copper Histidinate, for the Treatment of Menkes Disease
Rolling submission of New Drug Application to the FDA for CUTX-101 on track to begin in the first quarter of 2021 and to be completed by the end of the second
About this update from Fortress Biotech, Inc.
[{"type":"text","content":"Rolling submission of New Drug Application to the FDA for CUTX-101 on track to begin in the first quarter of 2021 and to be completed by the end of the second quarter of 2021\nNEW YORK, Dec. 15, 2020 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”), an innovative revenue-generating company focused on acquiring, developing and commercializing or monetizing promising biopharmaceutical products and product candidates cost-effectively, today announced that the U.S. Food and Drug Administration (“FDA”) has granted Breakthrough Therapy Designation to Cyprium Therapeutics (“Cyprium”) for CUTX-101, a potential treatment for Menkes disease. Often lethal if untreated, Menkes disease is an X-linked recessive disorder of copper metabolism caused by mutations in ATP7A, an evolutionarily conserved copper-transporting ATPase. The FDA previously granted Orphan Drug, Fast Track, and Rare Pediatric Disease Designations to CUTX-101 for the treatment of Menkes disease. Additionally, the European Medicines Agency previously granted Orphan Drug Designation to CUTX-101. “We are very pleased that the FDA has granted Breakthrough Therapy Designation to CUTX-101, a devastating pediatric disease with no FDA-approved treatment options currently available. The positive topline clinical efficacy data reported earlier this year highlight the potential of CUTX-101 to be a safe and effective therapy and fulfill a significant unmet medical need for patients with Menkes disease. We look forward to beginning our rolling submission of a New Drug Application (“NDA”) to the FDA for CUTX-101 in the first quarter of next year. Our goal is to bring this treatment to patients as soon as possible,” said Lung S. Yam, M.D., Ph.D., President and Chief Executive Officer of Cyprium. Breakthrough Therapy Designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for Breakthrough Therapy Designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. A Breakthrough Therapy Designation conveys all of the fast-track program features, more intensive FDA guidance on an efficient drug development program, an organizational commitment involving senior managers and eligibility fo...