Belief Pharmaceuticals Group has secured approval from the Macau Drug Administration for its gene therapy product BM-H901 injection (Popedacoq), for the treatment of adult patients with moderate to severe Haemophilia B.

The approval marks a significant milestone for the therapy, following its earlier clearance in mainland China in April 2025 as the country’s first gene therapy for haemophilia B. The latest nod expands access to patients in Macau and strengthens the global footprint of the therapy.

Developed and manufactured by Belief Pharmaceuticals, the product will be commercialised in mainland China, Hong Kong and Macau by Takeda China under a strategic partnership.

Haemophilia B is a hereditary disorder caused by a deficiency of clotting factor IX (FIX), requiring patients to undergo lifelong, frequent intravenous infusions of replacement therapies. The condition often leads to recurrent bleeding episodes, joint damage, disability and a significant economic burden.

Popedacoq is designed as a one-time gene therapy using a recombinant adeno-associated virus (rAAV) vector to deliver a functional FIX gene into liver cells. This enables sustained production of clotting factor IX, potentially reducing or eliminating the need for repeated injections.

Clinical data from multiple studies support the therapy’s efficacy and safety. In a Phase III trial involving 26 patients, the treatment significantly reduced annualised bleeding rates and decreased the need for FIX infusions. Patients also showed improved clotting factor levels and reduced joint bleeding episodes over a 52-week follow-up period.

Long-term data from earlier studies further demonstrated sustained benefits, with many patients maintaining therapeutic FIX levels for several years after a single dose. The therapy also showed a favourable safety profile, with no serious adverse events or inhibitor development reported.

The therapy has gained global regulatory recognition, including orphan drug designations from the U.S. Food and Drug Administration and classification as an advanced therapy medicinal product by the European Medicines Agency.

The approval in Macau is expected to accelerate broader international adoption and reflects growing support for innovative gene therapies in addressing rare genetic disorders.