WuXi Biologics Applauds CANbridge Pharmaceuticals for Milestone Approval of Velaglucerase-beta for Gaucher Disease Treatment

WuXi Biologics Applauds CANbridge Pharmaceuticals for Milestone Approval of Velaglucerase-beta for Gaucher Disease Treatment



WuXi Biologics, a prominent global Contract Research, Development, and Manufacturing Organization (CRDMO), has expressed congratulations to its partner CANbridge Pharmaceuticals following the approval of the innovative treatment, Velaglucerase-beta for injection, by the China National Medical Products Administration (NMPA). This approval marks a significant achievement, as it designates Velaglucerase-beta as the first locally developed Enzyme Replacement Therapy (ERT) for Gaucher disease in China.

A Breakthrough in Rare Disease Treatment



Gaucher disease, a relatively rare genetic disorder, has challenging symptoms including hepatosplenomegaly, anemia, bone pain, and neurological issues. These symptoms can become increasingly severe, making timely and effective treatment crucial. For over three decades, the standard treatment has been recombinant human glucocerebrosidase ERT, which has significantly enhanced patient outcomes and quality of life. It’s estimated that Gaucher disease affects 0.7 to 1.75 individuals per 100,000 worldwide, and it has been officially recognized on China’s First List of Rare Diseases.

Velaglucerase-beta has gone through an impressive journey from concept to commercial reality, thanks to WuXi Biologics' advanced integrated technology platform. This collaboration is significant not only for CANbridge but also for the landscape of rare disease therapeutics in China. The novel biologic has successfully passed all necessary manufacturing inspections, setting the stage for widespread accessibility and affordability for patients.

Innovative Technology Leading the Charge



One of the keys to the success of Velaglucerase-beta was WuXi Biologics’ use of cutting-edge technology to enhance production capabilities. By utilizing their WuXiUPTM platform, the team achieved over 110 times the overall yield and improved the specific enzyme activity by more than 50%. This innovation directly impacts the drug's cost-effectiveness, aiming to provide crucial support to patients managing their Gaucher disease.

Moreover, novel cell-based bioassay methods were crafted for both the product release and stability testing phases. This marks an advancement in ensuring product quality, addressing previous technical challenges associated with enzyme testing in ERT.

Industry Perspectives



Dr. Chris Chen, CEO of WuXi Biologics, shared his pride in enabling CANbridge to reach this pivotal milestone. He emphasized the collaboration's significance in the context of advancing drug innovation for rare diseases in China. Dr. Chen articulated that bridging the gap between early-stage discovery and patient access requires collective effort and commitment to addressing the unmet clinical needs surrounding rare diseases.

In a statement, Dr. James Xue, Founder and CEO of CANbridge Pharmaceuticals, recognized the relevance of enzyme replacement therapy in their global strategy. He expressed gratitude to all partners who contributed to this achievement, highlighting that the approval not only advances Velaglucerase-beta but also affirms CANbridge's dedication to developing rare disease therapies that are both accessible and effective.

Community Engagement and Future Initiatives



WuXi Biologics has also shown a strong commitment to fostering community awareness about rare diseases, engaging through various philanthropic initiatives. Their global “Run for Health” campaign and educational support for students affected by rare diseases in China are notable examples of their ongoing efforts to benefit patients and families facing these health challenges.

With 21 rare disease projects on their platform, WuXi Biologics continues to prioritize the development and manufacturing of innovative therapies, collaborating with global partners to enhance the quality of life for rare disease patients. As they look toward the future, their commitment to sustainability through green technology innovations underpins their business model, ensuring they deliver high-quality therapeutics while addressing broader societal needs.

In summary, the partnership between WuXi Biologics and CANbridge Pharmaceuticals represents a transformative step in the fight against Gaucher disease. The approval of Velaglucerase-beta not only serves the immediate therapeutic needs of patients but also sets a precedent for future collaborations aimed at developing impactful solutions for rare diseases globally.

Topics Health)

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