China’s Center for Drug Evaluation (CDE) has announced the inclusion of China Resources Double-Crane Pharmaceutical Co., Ltd’s (SHA: 600062) hydroxocobalamine and Sanofi (NASDAQ: SNY) Genzyme’s fitusiran in its patient-centered rare disease drug development pilot program, known as the Care Plan.
Hydroxocobalamine for Metabolic Disorders in Children with MMA
Hydroxocobalamine, also referred to as vitamin B12a, is utilized in the treatment of metabolic disorders in children affected by methylmalonic acidemia (MMA), with or without homocysteine. MMA is an autosomal recessive genetic disease primarily caused by defects in the methylmalonyl CoA mutase or its coenzyme cobalamin metabolism. The condition can be mitigated through vitamin B12 replacement therapy. The application of hydroxocobalamine for the Care Plan is categorized under Stage A, which marks the initiation of R&D projects. A comprehensive patient questionnaire survey will be conducted to inform the subsequent development and formulation of the drug, enhancing the understanding of MMA’s natural history.
Fitusiran for Hemophilia A and B Patients
Fitusiran is a once-monthly subcutaneously administered investigational RNA interference (RNAi) therapeutic that targets antithrombin (AT) to enhance thrombin generation (TG) and rebalance hemostasis in patients with hemophilia A (HA) or hemophilia B (HB), with or without inhibitors. The filing of fitusiran for the Care Plan is at Stage D, or the Pre NDA/NDA stage. A patient qualitative interview study is planned during clinical trials, with the aim of collecting and evaluating patient experience data related to safety, efficacy, and hemophilia treatment experience before and after using fitusiran during the post-marketing research phase.-Fineline Info & Tech
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