ABSTRACT
OBJECTIVE: To summarize the latest research progress in the development of enzyme replacement therapy drugs for rare diseases at home and abroad and their pharmaceutical assessment. METHODS: Based on the representative literature at home and abroad, this paper propose key points for consideration (such as molecular design, host selection, production process and quality control, etc.), as well as comments and suggestions for researcher of such products from review and evaluation perspective. RESULTS: Due to the complexity and heterogeneity of the drug molecules of enzyme replacement therapy for rare diseases, the quality research is the main difficulty in pharmaceutical development and evaluation. CONCLUSION: In recent years, in order to alleviate the lack of rare disease drugs in China and meet the urgent clinical needs, Chinese government has issued a series of policies to encourage the research and development of rare disease drugs.