Thoughts on common problems in pharmaceutical review of enzyme replacement therapy biologics for rare diseases
KAN Hong-jin
CHENG Su-yuan
Abstract：In recent years,the state has introduced a series of policies to encourage the development of rare disease drugs.This article analyzes the registration situation of enzyme replacement therapy biologics for rare diseases both domestically and internationally,the research and development challenges,summarizes the policy support information at the pharmaceutical research and development level,and discusses common technical issues in the pharmaceutical development of such products based on pharmaceutical review experience and relevant literature.
Keywords：rare diseasesenzyme replacement therapybiological products
Publication Date：2025-12-30
Online Publishing Date：2026-08-14(First online date of this platform, not the publication date of the document)
Pages：8( 2660-2667 )