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Mustang Bio Announces Rare Pediatric Disease Designation for MB-207 for the Treatment of X-linked Severe Combined Immunodeficiency in Previously Transplanted Patients
WORCESTER, Mass., Aug. 31, 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., Aug. 31, 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 Rare Pediatric Disease 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 were previously treated with a hematopoietic stem cell transplantation (“HSCT”) and for whom re-treatment is indicated.\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 Mustang’s Biologics License Application 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 or 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. Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, “XSCID is a life-threatening and rare genetic disorder with limited treatment options. We are pleased with our ongoing progress with the FDA to advance potential treatments for this devastating disease, including the recent Rare Pediatric Disease Designation for MB-107 in newly diagnosed XSCID patients and now for MB-207 for patients with XSCID who have previously received HSCT and require re-treatment. We look forward to continuing to work efficiently to expedite the development of critically needed treatment options for children with XSCID.” MB-207 is currently being assessed in a Phase 1/2 clinical trial for XSCID in patients over the age of two, who have received prior HSCT, at the National Institutes of Health. Mustang expects to file an investigational new drug application (“IND”) with the FDA to initiate a multi-center Phase 2 clinical trial of MB-207 in this patient population in the fourth quarter of 2020. Another Phase 1/2 clinical trial for XSCID in newly diagnosed infant...