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中生网 > 生物资讯 > 业界动态 > FDA提前批准治疗罕见儿童疾病新药Vimizim

FDA提前批准治疗罕见儿童疾病新药Vimizim

更新:2014年02月22日 阅读次数: 【字体:

 FDA提前批准治疗罕见儿童疾病新药Vimizim

2014年2月18日讯 /BIOON/ --美国时间2月14日下午,Biomarin Pharmaceutical 公司的新药Vimizim (elosulfase alfa) 被FDA正式批准,提前两周公布确实出人意料(原定于2月28日)。因FDA公布时间较晚,当日Biomarin 股票收盘价(75.78美元)并未受到影响。

Vimizim 是首个被FDA批准的治疗粘多糖沉积症 IVA型的药物。粘多糖病Ⅳ型(Morquio氏病),有两个亚型。其病因为ⅣA为半乳糖-6-硫酸酯酶(GALNS)缺乏,ⅣB为β-D半乳糖酶缺乏。该病为常染色体隐性遗传,其临床特点为明显的生长迟缓,步态异常和骨骼畸形且逐渐显著,病人寿命多为20~30岁。目前全美约有800名Morquio A综合症患者。

Vimizim 能够替代患者体内缺乏的GALNS酶。 通过176位病人的临床试验证实了该药物的安全性和有效性。依据FDA报告,其最常见的副作用包括发烧,头痛,恶心,腹痛和疲劳。(Bioon.com)

详细英文报道:

Vimizim (elosulfase alfa) has been approved by the U.S. Food and Drug Administration to treat a rare childhood disorder called Mucopolysaccharidosis Type IVA, also known as Morquio A syndrome.

The disorder is caused by a missing metabolic enzyme that leads to problems with bone development, growth and movement, the agency said in a news release. It affects about 800 people in the United States.

Vimizim replaces the missing enzyme, known as GALNS. The drug's safety and effectiveness were established in clinical trials involving 176 people, ranging in age from 5 to 57. The most common side effects included fever, vomiting, headache, nausea, abdominal pain, chills and fatigue, the FDA said.

The drug's safety and effectiveness weren't evaluated in children under age 5 years, the agency added. Vimizim's label will include a boxed warning to include the risk of anaphylaxis, an allergic-like reaction that could be life threatening.

Vimizim was the first drug granted a Rare Pediatric Disease Priority Review Voucher, an FDA effort to encourage development of new treatments for rare childhood diseases.

The drug is marketed by BioMarin Pharmaceuticals, based in Novoto, Calif.

 

 

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