Belief Pharmaceuticals Group has announced that its gene therapy BM-H901 injection (generic name: Popedacoq injection) has received approval from the Macau Drug Administration for treating adult patients with moderate to severe haemophilia B (congenital factor IX deficiency).

The therapy, developed and manufactured by Belief Pharmaceuticals, will be commercialized by Takeda China across mainland China, Hong Kong, and Macau. This approval follows its landmark clearance in mainland China in April 2025, where it became the country’s first approved gene therapy for haemophilia B.

The Macau approval marks a significant milestone, addressing the unmet need for gene therapy in the region and accelerating global access to this innovative treatment.

Dr. Xiao Xiao, co-founder, chairman, and chief scientist of Belief Pharmaceuticals, stated that the approval represents a key step in expanding cutting-edge gene therapy beyond mainland China. He highlighted that the therapy offers patients a transformative alternative aligned with international treatment standards, while also reflecting Macau’s progressive support for advanced medical technologies.

BM-H901 is designed as a one-time gene therapy that enables patients to continuously produce clotting factor IX (FIX) in the body. This approach has the potential to significantly reduce or eliminate the need for lifelong frequent injections associated with conventional therapies, thereby easing physical, psychological, and financial burdens on patients.

Haemophilia B is a hereditary bleeding disorder caused by a deficiency of clotting factor IX. Traditional treatments require frequent intravenous infusions of FIX or prothrombin complex concentrates, often leading to joint damage, disability, and increased risk of complications such as infections and thrombosis, along with high long-term treatment costs.

BM-H901 uses a recombinant adeno-associated virus (rAAV) vector to deliver a highly active FIX gene directly to liver cells. This enables sustained production of FIX in the bloodstream. The therapy incorporates an engineered liver-targeting capsid (AAV843) and the widely used FIX-Padua gene, along with a liver-specific promoter to ensure high and stable expression.

Clinical studies have demonstrated strong efficacy and safety outcomes. In a Phase III multicenter study involving 26 adult patients, the annualized bleeding rate (ABR) was reduced to 0.60 at 52 weeks, significantly lower than standard treatment benchmarks. Patients also experienced a substantial reduction in FIX infusions, from an average of 58.2 times per year before treatment to 2.9 times after treatment. Joint bleeding episodes were markedly reduced, with target joints eliminated.

Long-term data from an earlier investigator-initiated study showed sustained benefits over five years, with a majority of patients maintaining clinically meaningful FIX levels. Importantly, no serious adverse events, severe reactions, or inhibitor development were reported.

The therapy’s clinical findings have been published in leading international journals, including The Lancet Hematology, The New England Journal of Medicine, and Nature Medicine, and presented at major global forums such as the International Society on Thrombosis and Haemostasis (ISTH) Congress and the American Society of Hematology (ASH) Annual Meeting.

BM-H901 has also received multiple global recognitions, including Breakthrough Therapy designation in China, Orphan Drug Designation from the U.S. FDA, Rare Pediatric Disease Designation, Advanced Therapy Medicinal Product classification from the European Medicines Agency, and Orphan Drug Designation from Saudi Arabia.

Belief BioMed Inc. continues to focus on advancing gene therapy innovations for serious genetic and chronic diseases through its expertise in viral vector technology, with the goal of improving patient outcomes worldwide.