美国FDA批准拜玛林制药（BioMarinPharmaceutical）公司加硫酶（galsulfase）注射液(商品名：Naglazyme)上市,用于特异性治疗粘多糖病Ⅵ型(MPSⅥ)。由于本品是首个获准治疗MPSⅥ的药品，在美国处于罕用状态，享有7年的市场专卖权。

Naglazyme可改善患者行走和爬楼梯的能力。临床研究显示，本品对MPSⅥ患者具有重要的临床治疗意义，尤其是增加12分钟步行和3分钟爬楼梯的耐力。本品减少MPSⅥ患者尿中分泌过多的碳水化合物（葡糖胺聚糖，GAG），为酶活性的指标。

拜玛林制药公司完成了对39例MPSⅥ患者进行24周多中心双盲安慰剂对照的Ⅲ期临床研究。24周后，接受Naglazyme治疗的患者尿中GAG浓度较使用安慰剂者降低75.5%，在统计学上具有显著意义（P <0.001）。
规格:5MG/5ML,
NAGLAZYME VL 5MG DS 5ML
BIOMARIN PHARMACEUTICAL

Naglazyme® (galsulfase) for MPS VI

Naglazyme^® (galsulfase) is an enzyme replacement therapy for the treatment of mucopolysaccharidosis VI (MPS VI), an inherited life-threatening lysosomal storage disorder caused by a deficiency of the lysosomal enzyme
N-acetylgalactosamine 4-sulfatase. Naglazyme provides a recombinant version of this enzyme to individuals diagnosed with MPS VI.

Naglazyme at a Glance

• Approved in the United States, European Union, and at least 7 other countries in the rest of the world for the treatment of MPS VI
• Designated an orphan drug in the United States and internationally
• Manufactured and commercialized by BioMarin

Regulatory Status

In May 2005, Naglazyme received marketing approval from the US Food and drug Administration for the treatment of MPS VI patients. Shortly thereafter, in January 2006, Naglazyme received approval in the European Union. Naglazyme was designated an orphan drug in the US and EU, which grants market exclusivity for 7 and 10 years, respectively. Naglazyme has also been approved in Australia, Brazil, Croatia, Japan, South Korea, Switzerland, and Belarus. Naglazyme has been granted orphan drug designation in Australia, Japan, South Korea and Switzerland as well.

Commercialization Plan

BioMarin is commercializing Naglazyme worldwide.

Naglazyme is indicated for patients with mucopolysaccharidosis VI (MPS VI). Naglazyme has been shown to improve walking and stair-climbing capacity.