美国FDA批准埃洛磺酶α治疗罕见先天性酶疾病

• 作 者: 赵文丽
• 出 处: 国际药学研究杂志
• 发表日期: 2004年期
• 关键词: 埃洛 FDA 儿童疾病 硫酸酯酶 儿科疾病 乙酰半乳糖胺 常染色体隐性 代谢途径 溶酶体 综合征

美国FDA批准BioMarin公司的埃洛磺酶α(elosulfase alfa,Vimizim)上市,为首个用于治疗Morquio A综合征的药物。Morquio A综合征是一种常染色体隐性遗传性溶酶体贮存疾病,因N-乙酰半乳糖胺-6-硫酸酯酶(GALNS)缺乏引起。埃洛磺酶α可用来代替重要代谢途径中缺失的GALNS酶,缺乏此酶导致骨发育和生长问题。埃洛磺酶α是优先审评的罕见儿童疾病的首个药物,旨在鼓励可用于预防和治疗罕见儿科疾病

The FDA approved the launch of BioMarin's elosulfase alfa (Vimizim), the first drug to treat Morquio A syndrome. Morquio A syndrome is an autosomal recessive lysosomal storage disease caused by a deficiency of N-acetylgalactosamine-6-sulfatase (GALNS). Erosultine alpha can be used to replace the missing GALNS enzyme in important metabolic pathways, and lack of this enzyme leads to bone development and growth problems. Erlotin α is the first drug to be considered as a first-rate rare disease in children and is designed to encourage use in the prevention and treatment of rare pediatric diseases