Recently, according to the official website of CDE, Hydroxocobalamin Injection of China Resources Double Crane and Fitusiran Injection of Genzyme Corporation were included in the pilot project of the "Patient-centered Rare Disease Drug R&D Pilot Work Plan ("Care Plan")".

1. Hydroxocobalamin Injection

Variety name: Hydroxocobalamin injection
Applicant: China Resources Double Crane Pharmaceutical Co., Ltd.
Indications: This drug is suitable for the treatment of metabolic disorders in children with methylmalonic acidemia (MMA) with or without homocystinemia.
Application stage: Phase A - R&D project establishment stage.
Key points: During the R&D project establishment stage, detailed patient survey questionnaires will be collected to provide a basis for subsequent drug development, dosage forms, etc.; at the same time, the data on the natural history of MMA disease will be further enriched.

2. Fitusiran Injection

Product name: Fitusiran injection
Applicant: Genzyme Corporation
Indications: This product is indicated as a routine preventive treatment for adult patients with hemophilia A or B with or without inhibitors of coagulation factor VIII or IX and adolescent patients aged ≥12 years to prevent or reduce the frequency of bleeding.
Application stage: Stage D - pre-marketing application/marketing application stage.
Key points: It is planned to conduct qualitative patient interviews in clinical trials, and collect and evaluate patient experience data related to safety, efficacy, and hemophilia treatment experience before and after using fitusiran in the post-marketing research stage.

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