Sanofi's Agalsidase Beta (Fabrazyme) Approved in China as First Therapy for Fabry Disease

China’s National Medical Products Administration (NMPA) announced that it has recently approved the import registration application for agalsidase beta (Fabrazyme, trade name: Fabrazan), developed by Genzyme, a subsidiary of Sanofi. This enzyme replacement therapy is indicated for the treatment of Fabry disease, a rare genetic disorder. It is the first drug approved in China for the treatment of Fabry disease and is suitable for children aged 8 years and older, adolescents, and adults.

Fabry disease is a congenital metabolic disorder caused by X-linked genetic defects, characterized by significantly reduced or absent activity of α-galactosidase A in patients, leading to pathological accumulation of glycosphingolipids in the lysosomes of various tissue cells. The disease originates in the fetus and progressively worsens throughout the patient's life, causing failure of vital organs such as the heart and kidneys. In 2018, the disease was included in China's first batch of the Rare Disease Catalogue, classified as a condition that severely threatens life and for which there are currently no effective treatments available domestically.

Agalsidase Beta for Injection was submitted for registration by Sanofi (China) Investment Co., Ltd. as the agent for Genzyme Europe B.V. and was included in the second batch of the National List of Clinically Urgent New Drugs. In accordance with the relevant provisions of the "Announcement on Optimizing Drug Registration Review and Approval," the National Medical Products Administration (NMPA) included it in the priority review category. Based on overseas clinical trial data, combined with the efficacy and safety profile of the product, the NMPA approved its import registration application. The approval and market launch of this product will bring significant benefits to the treatment of Fabry disease patients in China.