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

China’s Center for Drug Evaluation (CDE) has released draft guidelines for the pharmaceutical common issues of recombinant adeno-associated virus (rAAV) vectors used in in vivo gene therapy products. The move aims to enhance R&D norms and accelerate the registration and high-quality development of domestic rAAV products.

Policy Breakdown

• Objective: The draft guidelines address key technical and regulatory questions to streamline the review process for gene therapy products.
• Consultation Period: Stakeholders have one month from the release date to submit feedback, reflecting the CDE’s commitment to inclusive regulation.
• Technical Focus: Guidelines cover critical aspects such as vector construction, quality control, and stability testing, providing clarity for developers navigating this complex space.

Market Implications
The initiative is expected to reduce regulatory uncertainty, potentially accelerating time-to-market for innovative gene therapies by 15–20%. Analysts at China Healthcare Innovation Institute highlight improved review predictability as a key driver for investment in the sector.

Industry Impact
Gene and cell therapy developers are encouraged to align with the emerging standards to ensure smoother registration processes. The guidelines underscore China’s push to establish itself as a global hub for advanced therapy development.

Looking Ahead
The finalized guidelines, expected by mid-2025, will likely shape the technical requirements for upcoming gene therapy applications. Stakeholders should actively engage in the consultation to influence the final framework.-China Health Reform Pulse

Policy Source: https://www.cde.org.cn/main/news/viewInfoCommon/fca7432d3e899f564e748f6c92238cac

China’s Center for Drug Evaluation (CDE) has launched a public consultation on its 2025 external expert roster, aiming to enhance the scientific rigor and impartiality of its drug review process. The move reflects the CDE’s ongoing efforts to build a transparent and credible regulatory framework.

Policy Breakdown

• Objective: The consultation, running from April 9 to April 17, seeks feedback on the proposed expert panel, which will support reviews of innovative and complex drug applications.
• Mechanism: Stakeholders can submit objections via email during the consultation period, ensuring accountability and public oversight.
• Regulatory Basis: The initiative adheres to the Interim Measures for the Management of External Experts of the National Medical Products Administration, emphasizing qualification reviews and procedural integrity.

Market Implications
The expanded expert panel is expected to accelerate reviews of high-priority applications, particularly for novel therapies and rare disease treatments. Analysts at China Healthcare Innovation Institute highlight improved review quality as a key benefit for both domestic and international developers.

Industry Impact
Pharmaceutical companies are encouraged to engage with the consultation process to ensure diverse expertise informs regulatory decisions. The move underscores China’s commitment to aligning its drug review practices with global standards.

Looking Ahead
The CDE plans to finalize the expert roster by mid-April, with the new panel expected to take effect immediately. Stakeholders should monitor how the expanded expertise influences review timelines and scientific guidance availability.-China Health Reform Pulse

Policy Source: https://www.cde.org.cn/main/news/viewInfoCommon/0bb5c2dd641e1438f164ad855460b852