China's Landmark Hemophilia Gene Therapy Secures Macao Approval, Eyeing Global Stage

SHANGHAI, China – March 19, 2026 – A groundbreaking gene therapy for hemophilia B has received regulatory approval in Macao, marking a significant milestone for patients in the region and for China's burgeoning biotech industry. Belief BioMed announced today that its single-dose treatment, BBM-H901 (Dalnacogene Ponparvovec Injection), has been greenlit by Macao's Pharmaceutical Administration Bureau.

The approval comes less than a year after the therapy became the first of its kind authorized in Mainland China in April 2025. For adult patients with moderate to severe hemophilia B, a debilitating genetic bleeding disorder, the news represents a potential end to a lifetime of frequent and burdensome injections, offering the promise of a functional cure in a single treatment.

A New Horizon Beyond Lifelong Injections

Hemophilia B is caused by a deficiency of a critical blood-clotting protein, Factor IX (FIX). For decades, the standard of care has been replacement therapy—regular intravenous infusions of manufactured FIX concentrate to prevent or treat bleeding episodes. While life-saving, this regimen imposes a heavy physical, psychological, and financial toll. Patients often face spontaneous bleeding into joints and muscles, leading to chronic pain, severe arthritis, and a high rate of disability.

BBM-H901 is designed to fundamentally change this reality. The one-time intravenous infusion has demonstrated remarkable success in clinical trials, with results published in prestigious journals like The New England Journal of Medicine and Nature Medicine. In a Phase 3 study involving 26 Chinese patients, the mean annualized bleeding rate (ABR) plummeted to just 0.6. This is a dramatic improvement over the average ABR of 5.0 seen in patients on standard prophylactic treatment.

Furthermore, the average number of FIX infusions required by participants dropped from 58 per year before the gene therapy to fewer than three per year afterward. Impressively, 80% of participants experienced no bleeding events at all in the year following treatment, and the number of painful "target joints" prone to bleeding was reduced to zero. Long-term data from an earlier trial is equally encouraging, with a five-year follow-up showing that 70% of participants maintained protective levels of Factor IX activity.

The Science of a Single-Dose Solution

Developed by Shanghai-based Belief BioMed, BBM-H901 harnesses advanced genetic engineering. The therapy uses a modified, harmless adeno-associated virus (rAAV) as a microscopic delivery vehicle. This vector is engineered with a special "capsid," or outer shell, called AAV843, which is designed to specifically target liver cells.

Once inside the liver, the vector delivers its payload: a functional copy of the Factor IX gene. This isn't just any version of the gene; it's the high-activity "FIX-Padua" variant, which produces a clotting factor that is many times more potent than the standard version. The patient's own liver cells are then transformed into bio-factories, continuously producing and releasing the crucial clotting protein into the bloodstream.

"BBM-H901 is a gene therapy designed to enable sustained and stable expression of coagulation factor IX in the body through a single administration," said Dr. Xiao Xiao, Co-founder and Chief Science Officer of BBM, in a statement. This approach, he noted, could free "patients from the physical, psychological, and economic burdens associated with lifelong frequent injections." The therapy also demonstrated a strong safety profile in trials, with no serious adverse events or the development of inhibitors, a common complication of traditional therapies.

Navigating a Complex Global Market

The approval in Macao is not just a clinical victory but a strategic one. Belief BioMed has partnered with Takeda China for the commercialization of BBM-H901 in Mainland China, Hong Kong, and Macao. The company sees Macao's status as an international hub as a key stepping stone for broader market access.

The global landscape for hemophilia gene therapy is both promising and fraught with challenges. While CSL Behring's Hemgenix has found a market in the U.S. and Europe since its 2022 approval, the path is not always smooth. In a cautionary tale for the industry, Pfizer announced in February 2025 that it was discontinuing its own recently approved hemophilia B gene therapy, Beqvez, citing limited interest from doctors and patients and concerns over long-term durability. High costs, with price tags running into the millions of dollars per dose, also remain a significant barrier to widespread adoption.

Against this backdrop, the successful rollout of BBM-H901 in Greater China could be pivotal. It provides a critical new option in a market with significant unmet need. While national registries list just under 4,000 hemophilia B patients in China, experts believe this represents less than 15% of the total, suggesting a large, underdiagnosed population that could benefit from advanced treatments.

China's Growing Influence in Advanced Therapies

The development and approval of BBM-H901 signals China's arrival as a major player in the highly complex field of gene therapy. The product has already garnered international recognition, receiving Orphan Drug Designation from regulatory bodies in the U.S. and Saudi Arabia, as well as an Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency. These designations acknowledge the therapy's innovative nature and potential to address a rare, serious disease.

By achieving these back-to-back approvals in Mainland China and Macao, Belief BioMed has demonstrated an ability to navigate complex regulatory pathways and produce a therapy that meets global standards. The collaboration with a multinational pharmaceutical giant like Takeda further validates the technology and provides a powerful channel to bring this Chinese innovation to patients. As BBM-H901 becomes available, it will not only transform lives but also serve as a powerful symbol of a new era in global biotechnology, where breakthrough innovations are emerging from all corners of the world.