Fortress Biotech Announces Positive Opinion on Orphan Drug Designation Received from the European Medicines Agency for CUTX-101, Copper Histidinate, for the Treatment of Menkes Disease
NEW YORK, July 31, 2020 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”), an innovative biopharmaceutical company focused on acquiring, developing and commercializing high-potential marketed pharmaceutical products and development-stage pharmaceutical product candidates, today announced that the European Medicines Agency (“EMA”) Committee for Orphan Medicinal Products issued a positive opinion on Cyprium Therapeutics’ (“Cyprium”) application for Orphan Drug Designation for Copper Histidinate, also referred to as CUTX-101, a potential treatment for Menkes disease. Menkes disease is an often lethal, if untreated, X-linked recessive disorder of copper metabolism caused by mutations in ATP7A, an evolutionarily conserved copper-transporting ATPase. The U.S. Food and Drug Administration (“FDA”) previously granted Orphan Drug, Fast Track and Rare Pediatric Disease Designations to CUTX-101 for the treatment of Menkes disease.
Lung S. Yam, M.D., Ph.D., President and Chief Executive Officer of Cyprium, said, “The positive opinion for Orphan Drug Designation from the EMA is an important milestone in bringing a much-needed potential therapy to patients with Menkes disease, a devastating pediatric disease with limited treatment options. We look forward to working closely with the EMA and continuing to progress CUTX-101 for children in need. To this end, we remain on track to begin a rolling submission of a New Drug Application to the FDA for CUTX-101 in the U.S. in the fourth quarter of this year.”
Orphan Drug Designation in the European Union (“EU”) is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products. To qualify, an investigational medicine must be intended to treat a seriously debilitating or life-threatening condition that affects fewer than five in 10,000 people in the EU, and there must be sufficient non-clinical or clinical data to suggest the investigational medicine may produce clinically relevant outcomes. EMA orphan drug designation provides companies with certain benefits and incentives, including clinical protocol assistance, differentiated evaluation procedures for Health Technology Assessments in certain countries, access to a centralized marketing authorization procedure valid in all EU member states, reduced regulatory fees and 10 years of market exclusivity.
CambPharma Solutions (CY) Limited submitted the Orphan Drug Designation application on behalf of Cyprium, as its agent in the EU.
About Menkes Disease and Related Copper Metabolism Disorders
About CUTX-101 (Copper Histidinate)
About Cyprium Therapeutics
About Fortress Biotech
Lung Yam, M.D., Ph.D.
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