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Mustang Bio Announces Positive Opinion from the European Medicines Agency on Orphan Drug Designation for Its Lentiviral Gene Therapy for the Treatment of X-linked Severe Combined Immunodeficiency (“XSCID”)
WORCESTER, Mass., Nov. 24, 2020 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (“Mustang”) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on
About this update from Mustang Bio, Inc.
[{"type":"text","content":"WORCESTER, Mass., Nov. 24, 2020 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (“Mustang”) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that the European Commission (“EC”) issued a positive opinion on its application for Orphan Drug Designation for Mustang’s lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency (“XSCID”), also known as bubble boy disease. The Designation applies both to MB-107 for the treatment of newly diagnosed infants between two months and two years of age and to MB-207 for the treatment of patients who have been previously treated with hematopoietic stem cell transplantation (“HSCT”) and for whom re-treatment is indicated. The European Medicines Agency (“EMA”) previously granted Advanced Therapy Medicinal Product classification to MB-107 in April 2020. The U.S. Food and Drug Administration (“FDA”) also previously granted Rare Pediatric Disease and Orphan Drug Designations to MB-107 and MB-207, as well as Regenerative Medicine Advanced Therapy Designation to MB-107.\n Orphan Drug Designation in the European Union (“EU”) is granted by the European Commission based on a positive opinion issued by the European Medicines Agency Committee for Orphan Medicinal Products (EMA COMP). 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 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. Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, “We are very pleased to receive a positive opinion from the EC on Orphan Drug Designation for our lentiviral gene therapy for XSCID. It is an important milestone for Mustang as we...