Mustang Bio Receives Advanced Therapy Medicinal Product Classification
Mustang Bio, Inc. recently announced that the European Medicines Agency (EMA) has granted Advanced Therapy Medicinal Product (ATMP) classification to MB-107, Mustang’s lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease. The US FDA previously granted Regenerative Medicine Advanced Therapy (RMAT) designation to MB-107 for the treatment of XSCID in August 2019.
EMA grants ATMP classifications to new therapeutics that are based on genes or cells and intended as long-term or permanent therapeutic solutions to acute or chronic human diseases at a genetic, cellular, or tissue level. The ATMP program provides specific regulatory guidelines for preclinical development, manufacturing, and product quality testing of ATMPs and offers incentives, including fee reductions for regulatory advice, recommendations, and evaluation and certification of quality and non-clinical data.
Manuel Litchman, MD, President and Chief Executive Officer of Mustang, said “We are extremely encouraged that the EMA has granted MB-107 with ATMP classification, an important step in establishing our path to market approval and commercialization in Europe. This classification complements the RMAT designation we received last year from the FDA and brings us closer to realizing our goal of commercializing MB-107 for XSCID patients, as these patients are in desperate need of innovative and potentially curative treatment options.”
MB-107 is currently being assessed in two Phase 1/2 clinical trials for XSCID: the first in newly diagnosed infants under the age of 2 at St. Jude Children’s Research Hospital (St. Jude), UCSF Benioff Children’s Hospital in San Francisco and Seattle Children’s Hospital and the second in patients over the age of 2 who have received prior hematopoietic stem cell transplantation at the National Institutes of Health. Under a licensing partnership with St. Jude, Mustang intends to develop the lentiviral gene therapy for commercial use as MB-107.
Mustang Bio, Inc. is 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. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to out-license or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for XSCID. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the US Securities and Exchange Commission. Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit www.mustangbio.com.
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