Belief BioMed (BBM) and Takeda China have announced China’s National Medical Products Administration’s (NMPA) approval for BBM-H901 (Dalnacogene Ponparvovec) injection, a gene therapy for adults with moderate to severe haemophilia B, a bleeding disorder.
This is the first such therapy approved for haemophilia B in the country.
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Developed and manufactured by BBM, the therapy is based on a recombinant adeno-associated virus vector and delivers a human coagulation factor IX (FIX) gene into the patient’s liver cells.
The host cell’s gene transcription system then enables continuous expression and secretion of coagulation FIX into the bloodstream, supporting coagulation.
The collaboration between BBM and Takeda China aims to leverage their resources to expedite the therapy’s availability to sufferers.
Takeda China handles the therapy’s commercialisation in mainland China, Macau and Hong Kong.
BBM chairman, co-founder and chief science officer Dr Xiao Xiao stated: “The approval of BBM-H901 is an important milestone in the development of BBM. As the first haemophilia B gene therapy drug independently developed in China, BBM-H901 will provide a new treatment option for haemophilia B patients, which is expected to provide patients with an innovative treatment solution and help them return to a normal life.
“BBM will work together with Takeda China to accelerate the rapid implementation of this innovative therapy. In the future, BBM will strive to accelerate the overseas commercialisation process of BBM-H901, hoping to benefit more haemophilia B patients. “
In 2024, the therapy received US Food and Drug Administration (FDA) rare paediatric disease designation and the European Medicines Agency’s (EMA) Advanced Therapy Medical Products designation.
It secured the investigational new drug application approval in China in August 2021, and holds FDA orphan drug designation.
In September 2024, Asklepios BioPharmaceutical entered a strategic partnership with BBM to progress the development of new gene therapies.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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