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Mustang Bio Announces Orphan Drug Designation for MB-207 for the Treatment of X-linked Severe Combined Immunodeficiency (“XSCID”) in Previously Transplanted Patients
WORCESTER, Mass., Sept. 22, 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., Sept. 22, 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 U.S. Food and Drug Administration (“FDA”) has granted Orphan Drug Designation to MB-207, Mustang’s lentiviral gene therapy for the treatment of patients with X-linked severe combined immunodeficiency (“XSCID”), also known as bubble boy disease, who have been previously treated with a hematopoietic stem cell transplantation (“HSCT”) and for whom re-treatment is indicated. In August 2020, the FDA granted Rare Pediatric Disease Designation to MB-207 for the treatment of XSCID in previously treated patients with HSCT.\n The FDA grants Orphan Drug Designation to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain incentives, such as tax credits toward the cost of clinical trials and prescription drug user fee waivers. If a product holding Orphan Drug Designation receives the first FDA approval for the disease in which it has such designation, the product is entitled to seven years of market exclusivity, which is independent from intellectual property protection. Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, “The receipt of Orphan Drug and Rare Pediatric Disease Designations from the FDA for MB-207, to treat XSCID patients who have received a prior HSCT and for whom re-treatment is necessary, are important regulatory milestones for Mustang that are indicative of the significant unmet need in this devastating rare disease. These achievements bring us closer to providing a new potential treatment option to patients and their families. We look forward to initiating our pivotal clinical programs for MB-207 in previously transplanted patients with XSCID and MB-107 in newly diagnosed infants with XSCID.” The lentiviral gene-therapy method employed in MB-207 was co-developed by scientists at the National Institute of Allergy and Infectious Diseases (“NIAID”), part of the National Institutes of Health, and St. Jude Ch...