2022年12月21日，信念医药公司（Belief BioMed，BBM）（下称“信念医药”）全资子公司上海信致医药科技有限公司宣布，其自主研发和生产的BBM-H803注射液，获得美国食品药品监督管理局（FDA）的孤儿药认定（ODD），用于治疗血友病A，这是信念医药第二款获得FDA孤儿药认定的基因治疗药物。第一款用于治疗血友病B的基因治疗药物（BBM-H901注射液）已于今年8月15日获得FDA的孤儿药认定。
BBM-H803注射液是信念医药拥有自主知识产权的AAV基因治疗药物，通过静脉给药将人凝血因子Ⅷ（Factor Ⅷ，FⅧ）基因导入血友病A患者体内，从而提高并长期维持患者体内凝血因子水平，以期达到“一次治疗、长期有效”的治疗及预防出血效果。BBM-H803注射液药物的设计采用了拥有完全自主知识产权的肝靶向性血清型和高效基因表达盒，运用公司自主开发的无血清悬浮培养和层析工艺进行符合GMP要求的药物生产。根据FDA官网信息，获得FDA孤儿药认定后，申请人可以享受如下政策红利：
1)税收抵免，高达临床开发的50%（Tax credits for qualified clinical trials）
2)免除申请费用：可免除BLA/NDA的User fee（Exemption from user fees）
3)FDA批准后的7年市场独占期（Potential seven years of market exclusivity after approval）此外，申办者还能得到FDA在开发过程中的协助（Assistance in the drug development process ）以及孤儿药产品补助资金（Orphan Products Grant funding）。信念医药成立于2018年，是一家立足全球的创新生物医药公司，专注于开发以腺相关病毒（Adeno-associated virus, AAV）为载体的创新型基因疗法，已成为全球领先的覆盖早期研究到商业生产的基因治疗一体化开发平台。信念医药旨在为基因缺陷遗传疾病、老年退行性疾病以及其它重大恶性疾病提供更加有效、安全的疗法。信念医药除了拥有创新性的AAV衣壳和表达盒，在工艺和质检方面也取得了多项自主开发和创新，形成了一套先进、可靠、高效的CMC工艺流程，单次生产体积可达500L。信念医药的研发和生产实力获得顶尖投资机构及企业的认可，公司已在中国上海、香港、北京和苏州以及美国北卡罗纳州设立了办公室或研发、生产中心。更多信息请访问：www.beliefbiomed.com
Belief BioMed’s Second AAV-based Gene Therapy Drug Candidate was Granted Orphan Drug Designation by the FDABelief BioMed (BBM, Company) announced today that its self-developed and manufactured BBM-H803 injection was granted orphan drug designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of hemophilia A on December 21, 2022. It is the second gene therapy drug candidate by the Company (via its wholly owned subsidiary, Shanghai Belief-Delivery BioMed Co., Ltd.) to have obtained FDA orphan drug designation following the BBM-H901 injection for hemophilia B on August 15, 2022.BBM-H803 Injection is a recombinant adeno-associated virus (AAV) based gene therapy drug for hemophilia A developed and manufactured by Belief BioMed, containing a codon-optimized human factor Ⅷ gene under the control of a liver-specific promoter. Belief BioMed owns the proprietary patents of the capsid and the gene cassette. Leveraging the serum-free production and chromatographic purification process developed in-house by Belief BioMed to produce drugs in line with cGMP requirements, BBM-H803 has demonstrated high efficacy and safety with the FⅧ (Factor Ⅷ) well-expressed in patient’s plasma, achieving “one single treatment, long-term benefit” effect.FDA Orphan Drug Designation PolicyAfter obtaining the FDA orphan drug designation, it qualifies the sponsors for incentives, including:1)Tax credits for qualified clinical trials, up to 50% of clinical development,2)Exemption from user fees: User fee waiver for BLA/NDA,3)Potential seven years of market exclusivity after approvalIn addition, sponsors receive FDA assistance in the drug development process and Orphan Products Grant funding.Belief BioMed is aspiring to become a globally leading gene therapy company by being committed to providing innovative therapies with improved efficacy for monogenic disorder diseases, age-related degenerative diseases, and certain malignant diseases through its AAV vector technology from early discovery to commercialization. BBM has the proprietary rights of AAV capsids and expression cassettes and has achieved numerous proprietary improvements in process and quality control, forming an advanced, reliable, efficient manufacturing process with a single production cell culture volume of up to 500L currently. The R&D and production strengths of Belief BioMed have been recognized by top investment institutions and enterprises. Belief BioMed has offices, research centers and manufacturing facilities in Shanghai, Hong Kong, Beijing and Suzhou, China and North Carolina, US.Please visit www.beliefbiomed.com for more information