Genzyme公司向FDA递交Myozyme的上市申请

Genzyme公司称，已经向FDA递交了Myozyme药物的上市申请以及表明其在治疗酸性麦芽糖酶缺乏症（Pompe病）方面达到临床主要分析指标的最新试验数据。 Pompe病是由于遗传上的酶缺失而导致的一种使人虚弱无力甚至致命的肌肉疾病，全球的患者少于1万人。目前还没有一种治疗方法通过批准。 Genzyme已经获得FDA的罕用药证书，这意味着一旦药物获得 FDA通过，将享有7年的市场独占权。去年12月，Genzyme向欧盟提出了申请，还计划今年晚些时候在日本提出申请。

Genzyme Corp. said Friday that it submitted a marketing application for Myozyme to the Food and Drug Administration along with just-released clinical trial results showing the treatment for Pompe disease met its primary endpoint.Pompe disease is a debilitating and often fatal muscle disorder that affects less than 10,000 people worldwide, caused by an inherited enzyme deficiency. There is no approved treatment for the disease, the company said.

Genzyme released results from a clinical trial that tested the treatment in infants who began showing signs of the disease. In a study that compared 18 infants given Myozyme with historical results of infants with the disease, researchers found that 83 percent of infants given Myozyme were alive and not using a mechanical respirator after 18 months, compared with the 2 percent of infants who survived to 18 months in the other group.

The company said it expects the FDA to give the drug a priority review, meaning the agency will decide whether or not to approve the drug in six months rather than the standard 10 months. The company has already received an orphan drug designation from the FDA, meaning Genzyme will get seven years of marketing exclusivity if the drug is approved.

Genzyme applied for European approval in December, and expects to file in Japan later this year.

The company's shares fell 47 cents to $74.73 in morning trading on the Nasdaq.

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