Shanghai, China, December 15, 2025 — Belief BioMed (BBM), an innovative biotechnology company focused on developing cutting-edge gene therapies, today announced that the Saudi Food and Drug Administration (FDA) has recently granted Orphan Drug Designation (ODD) to the company’s gene therapy drug BBM-H901 for the treatment of patients aged 12 years and older with moderate to severe hemophilia B (congenital coagulation factor IX deficiency).

BBM-H901 (generic name: Dalnacogene Ponparvovec), has been officially approved by the National Medical Products Administration (NMPA) of China for the treatment of adult patients with moderate to severe hemophilia B (congenital coagulation factor IX deficiency) on April 8, 2025, and obtained the Breakthrough Therapy Designation by the Center for Drug Evaluation, NMPA in 2022. Previously, BBM-H901 was granted ODD by U.S. FDA in 2022, and was granted Rare Pediatric Disease Designation (RPDD) by U.S. FDA and Advanced Therapy Medicinal Product (ATMP) Designation by European Medicines Agency (EMA). This ODD designation of SFDA indicates that BBM-H101 has been positively acknowledged for its potential clinical value.

ODD is a special policy introduced by SFDA to encourage the development of therapies for rare diseases. Products granted ODD benefit from expedited review, regulatory-strategy guidance, and other supportive measures. After obtaining ODD, BBM-H901 will be submitted directly as a New Drug Application (NDA), potentially shortening the approval timeline and bringing the therapy to patients in the Middle East sooner.

About Hemophilia B (HB)

Hemophilia B is an inherited bleeding disorder caused by the deficiency of factor IX (FIX)¹. For a long time, patients can only rely on prothrombin complex concentrate (PCC) or FIX as a replacement therapy^2,3. Persistent and frequent bleeding can easily lead to damage of joint structure and function, resulting in a high disability rate⁴. This not only brings great physical pain and inconvenience to patients, but also comes with the risk of infection, blood clots, etc.^2,3. The high medical treatment costs also bring a heavy and constant economic burden on the patient's family.

About BBM-H901

BBM-H901 (generic name: Dalnacogene Ponparvovec), has been officially approved by NMPA for the treatment of adult patients with moderate to severe hemophilia B (congenital coagulation factor IX deficiency) on April 8, 2025, and it is also the first approved hemophilia B gene therapy in China. BBM-H901 is developed and manufactured by BBM. It can deliver the optimized human coagulation FIX gene into liver cells of patients to express continuously, and thus the patient's coagulation FIX level is improved and maintained. In 2022, the research results of IIT were successively published in two authoritative international journals, The Lancet-Hematology⁴ and The New England Journal of Medicine⁵. In the same year, BBM-H901 obtained the Breakthrough Therapy Designation by the Center for Drug Evaluation, NMPA. In 2024, the long-term follow-up results of more than 3 years of IIT were orally presented at the 2024 International Society on Thrombosis and Haemostasis (ISTH) Congress, and the results of the Phase 3 clinical study were released by BBM at the 66th Annual Meeting of the American Society of Hematology (ASH)⁶. In 2025, the long-term follow-up results of IIT were orally presented at the 2025 ISTH Congress again⁷, and the 2-year long-term follow-up results of the registration trial were presented at the Asia Pacific Thrombosis and Haemostasis Conference (APSTH)⁷. In the same year, the clinical trial results of BBM-H901 were published in the authoritative international journal Nature Medicine⁸.

References

1. Thrombosis and Hemostasis Group, Chinese Society of Hematology, Chinese Medical Association / Hemophilia Treatment Center     Collaborative Network of China. Chinese guidelines on the treatment of hemophilia (version 2020). Chin J Hematol.2020;41(4):265-271

2. Mortensen GL,et al. Haemophilia. 2018;862–872

3. Srivastava A, et al. Haemophilia. 2020;001–158.P95

4. Xue F, et al. Lancet Haematol. 2022 Jul;9(7):e504-e513

5. Xue F, et al. N Engl J Med. 2022 Oct 27;387(17):1622-1624

6. Feng X, et al. 2024 ASH. Poster 3582

7. Mankai Ju, et al. 2025 ISTH. OC 69.2

8. Xue F, et al. 2025 APSTH. Poster-48

9. Xue F, et al. Nat Med. 2025 Nov