Core Viewpoint - The approval of the injection drug Velaglucerase beta (brand name: Gorynin) by the National Medical Products Administration (NMPA) marks a significant milestone as it is the first domestically developed enzyme replacement therapy for Gaucher disease in China, targeting adolescents and adults aged 12 and above with Type I and III Gaucher disease [1][2]. Group 1: Product Approval and Significance - Velaglucerase beta is the only locally developed enzyme replacement therapy for Gaucher disease in China, making it a unique addition to the treatment options available [1]. - The drug is noted for having the broadest range of indications among Gaucher disease treatments currently available [1]. Group 2: Disease Background and Treatment - Gaucher disease is a rare genetic lysosomal storage disorder, with clinical manifestations including hepatosplenomegaly, anemia, bone pain, and neurological complications, potentially leading to life-threatening conditions [2]. - The global prevalence of Gaucher disease is estimated at 0.7 to 1.75 cases per 100,000 people [2]. - Enzyme replacement therapy (ERT) has been the standard treatment for Gaucher disease for 30 years, showing significant efficacy in improving non-neurological symptoms and enhancing the quality of life for patients [2]. Group 3: Development and Technology - The development of Gorynin was facilitated by collaborative efforts with partners like WuXi Biologics, which helped reduce development costs and ensure the drug is safe and effective for patients [2]. - The drug's development utilized second-generation innovative technology, resulting in over a 110-fold increase in production yield and more than a 50% enhancement in enzyme activity, significantly lowering production costs [2]. - WuXi Biologics also developed a cell-based activity assay for quality control, addressing the unique challenges of testing enzyme-based drugs [2].
国内首个本土创新戈谢病酶替代疗法获批上市