Group 1 - The approval of the drug "Gorainin" marks the first domestically developed enzyme replacement therapy for Gaucher disease in China, addressing a significant unmet medical need for patients who previously relied on imported treatments [1][2] - Gaucher disease is a rare genetic lysosomal storage disorder characterized by symptoms such as hepatosplenomegaly, anemia, bone pain, and neurological complications, with severe cases posing life-threatening risks [1] - The drug is specifically indicated for adolescents and adults aged 12 and above with Type I and Type III Gaucher disease, providing a targeted treatment option through intravenous infusion [1] Group 2 - The annual treatment cost for patients using imported drugs ranges from 1.5 million to 2.5 million RMB, making it unaffordable for many, highlighting the need for local production to improve accessibility [2] - The company aims to reduce the annual treatment cost of Gorainin by over 50% compared to imported alternatives, enhancing affordability for patients [2] - The drug's development involved collaboration with WuXi Biologics, utilizing innovative technology to significantly increase production yield and enzyme activity, ensuring better availability for patients [3][5] Group 3 - The drug is part of a pilot project for segmented production of biopharmaceuticals in China, which allows for flexible resource allocation and cost reduction in manufacturing [4][5] - The segmented production model has been successfully implemented for Gorainin, with the active ingredient produced by WuXi Biologics and the formulation handled by its subsidiary, demonstrating a new approach in the industry [5]
首个国产戈谢病酶替代疗法获批上市，价格有望比进口药低一半以上