Belief BioMed’s Second AAV-based Gene Therapy Drug Candidate was Granted Orphan Drug Designation by the FDA
Shanghai, CN, Orange, NC December 22, 2022
Belief 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.
Source: FDA website
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 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.