启明星 | 信念医药针对血友病B的基因疗法获得欧洲药品管理局先进治疗药物认证 | Bilingual

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日前，启明创投投资企业、专注前沿基因治疗领域的全球化创新型企业信念医药集团（Belief BioMed，BBM，下称“信念医药”）宣布，欧洲药品管理局（EMA）授予其基因治疗药物BBM-H901注射液先进治疗药物分类认证（Advanced Therapy Medicinal Product, ATMP），用于治疗血友病B。

BBM-H901注射液由信念医药自主研发和生产，是一款携带优化人凝血因子Ⅸ（FIX）基因表达盒的重组腺相关病毒（rAAV）基因治疗药物。欧洲药品管理局作为制定全球药品监管标准的重要力量，授予信念医药该项认证，意味着这一突破性研发成果再次获得监管机构的肯定，公司亦将藉此进一步推进全球化战略以及获得欧洲市场认可。

根据欧盟规定，EMA一般将ATMP授予基于基因、组织或体细胞的疗法，这些疗法有望为人类疾病或损伤带来突破性的治疗方案。通过ATMP的主要权益包括：EMA为认证产品的开发、临床以及生产提供一系列监管指南和激励措施，其中还包括降低科学咨询等相关费用。  

作为信念医药的重磅产品之一，BBM-H901注射液已经取得诸多瞩目成绩，其临床潜力与学术价值得以持续印证和彰显：该款药物先后被纳为中国国家药品监督管理局（NMPA）突破性治疗药物（BTD）品种和新药上市申请（NDA）优先审评品种；获得美国食品药品监督管理局（FDA）的孤儿药认定（ODD）以及儿科罕见病资格认定（RPDD）；研究者发起的临床研究（IIT）结果发表于国际权威期刊《柳叶刀-血液病学》和《新英格兰医学杂志》，并在2024国际血栓与止血学会（ISTH）大会进行展示。

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.