China’s National Medical Products Administration (NMPA) has approved Shanghai BeliefDelivery BioMed Co Ltd’s BBM-H901 injection (brand name: Xinjiu Ning) for the treatment of adult patients with moderate to severe hemophilia B. The move highlights China’s commitment to advancing gene therapy and providing new treatment options for rare diseases.
Policy Breakdown
- Approval Mechanism: The drug was approved through an expedited review process, reflecting the NMPA’s focus on accelerating access to innovative therapies.
- Therapeutic Innovation: BBM-H901 is a recombinant adeno-associated virus (rAAV) vector gene therapy product that delivers the FIX gene to target cells (primarily liver cells) to express FIX.
- Market Implications: The approval offers a new treatment paradigm for hemophilia B patients, potentially reducing the burden of frequent infusions and improving quality of life.
Market Implications
The approval signals China’s growing role as a hub for gene therapy innovation. Analysts at China Healthcare Innovation Institute estimate that the market for hemophilia treatments could expand by 15–20% with the introduction of gene therapies.
Industry Impact
Pharmaceutical companies are encouraged to leverage China’s supportive regulatory environment to advance similar therapies. The move underscores the government’s push for high-quality development in the pharmaceutical sector, as outlined in the 14th Five-Year Plan for National Drug Safety and High-Quality Development.
Looking Ahead
Stakeholders should monitor how the NMPA’s expedited review pathways and gene therapy guidelines evolve to support further innovation. The long-term goal is to create a more conducive environment for the development and commercialization of advanced therapies.-China Health Reform Pulse
Policy Source: https://www.nmpa.gov.cn/zhuanti/cxylqx/cxypxx/20250410095314169.html