The gene therapy for hemophilia B developed by Belief BioMed has received Advanced Therapy Medicinal Product Designation from the EMA
Our portfolio company, Belief BioMed Inc. (BBM), a globally innovative biotech company focusing on cutting-edge gene therapies, announced that the European Medicines Agency (EMA) has granted its gene therapy drug BBM-H901 the Advanced Therapy Medicinal Product (ATMP) designation for the treatment of hemophilia B.
BBM-H901, independently developed and manufactured by Belief BioMed, is a recombinant adeno-associated virus (rAAV) gene therapy vector carrying an optimized human Factor IX (FIX) gene expression cassette. This EMA designation, from one of the leading global regulatory authorities, validates its breakthrough research, advances its global expansion plans, and strengthens its position in the European market.
In accordance with EU regulations, the EMA typically designates Advanced Therapy Medicinal Products (ATMPs) to therapies based on genes, tissues, or somatic-cells that are anticipated to provide transformative treatment options for human diseases or injuries. ATMP developers can receive a range of regulatory guidelines for pre-clinical research, clinical development and manufacturing, as well as incentives, such as fee reduction for scientific advice.
As one of Belief BioMed's core products, BBM-H901 has received a lot of recognition for its clinical potential and academic significance: It has received a Breakthrough Therapy Designation (BTD) and was granted Priority Review under the New Drug Application (NDA) by the National Medical Products Administration (NMPA) of China; it has also obtained the Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the US Food and Drug Administration (FDA); results from Investigator Initiated Trial (IIT) related to BBM-H901 have been published in prestigious international journals such as The Lancet Haematology, The New England Journal of Medicine, and presented at the International Society on Thrombosis and Haemostasis (ISTH) 2024 Congress.
