Agarase β for injection of drugs for rare diseases is approved for marketing

Recently, the National Drug Administration approved Genzyme Europe B.V.'s application for the import registration of agatase β for injection (trade name: fabuzen) The drug is the first approved drug for treating Fabry disease in China, which is suitable for children, adolescents and adults over 8 years old Fabry's disease is a kind of congenital metabolic disease caused by X-linked gene defects, which is manifested in the obvious decrease or absence of α - galactosidase a activity in patients, resulting in pathological accumulation of glycosphingolipids in lysosomes of various tissue cells The disease begins in the fetus and gradually deteriorates with the patient's life, resulting in the failure of heart, kidney and other important organs In 2018, the disease was included in the first batch of national rare diseases catalogue, which is a serious life-threatening disease and there is no effective treatment in China The approved enzyme β for injection was declared by Sanofi (China) Investment Co., Ltd on behalf of Genzyme Europe B.V., and was listed in the second batch of new drugs urgently needed in clinical practice In accordance with the relevant provisions of the announcement on optimizing the review and approval of drug registration, the State Food and drug administration shall include it into the priority review varieties, and approve its import registration application based on the overseas clinical trial data and in combination with the effectiveness and safety characteristics of the product The approval of this product will bring good news for the treatment of domestic patients with Fabry disease Adorable adorable