Belief BioMed’s AAV-based Gene Therapy Drug Candidate was Granted Orphan Drug Designation by the FDA
Shanghai, CN, Orange, NC August 17, 2022
Belief BioMed (BBM, Company) announced today, that its self-developed and manufactured BBM-H901 injection has been granted orphan drug designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of hemophilia B. It is the first gene therapy drug by the Company (via its wholly owned subsidiary, Shanghai Belief-Delivery BioMed Co., LTD.) to have obtained FDA orphan drug designation.
Source: FDA website
BBM-H901 is recombinant adeno-associated virus (AAV) based gene therapy drug for hemophilia B developed and manufactured by Belief BioMed, containing a codon-optimized human factor IX 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-H901 has demonstrated high efficacy and safety with the FIX (Factor IX) well-expressed in patient’s plasma, achieving “one single treatment, long-term benefit” effect.
As the Investigator Initiated Trial (NCT04135300) for BBM-H901 was started in 2019, the early phase clinical trial results have been published in The Lancet Hematology, a leading international journal in hematology, in May 2022. At the same time, the product was approved for registered clinical trials (CTR20212816) in August 2021, and the first subject was dosed on December 30, 2021. At present, the multi-center, single-arm, open-label, single-dose clinical study of the product is being accelerated. The clinical research data of BBM-H901 injection have shown good safety and efficacy. After AAV gene therapy drug infusion, patients' Annualized Bleeding Rate (ABR) was significantly reduced, and the levels of coagulation factors in the body significantly increased with long-term stable expression. The safety profile was good, and no serious adverse events were reported.
FDA Orphan Drug Designation Policy
After 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 approval
In addition, sponsors receive FDA assistance in the drug development process and Orphan Products Grant funding.
About Belief BioMed
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.