Genzyme: Myozyme Receives Positive Opinion from European Regulatory Committee
Proposed Indication Includes All Patients with Pompe Disease
Genzyme Corp. announced that the Committee for Human Medicinal Products (CHMP) of the European Medicines Agency has adopted a positive opinion on the marketing authorization application for Myozyme® (alglucosidase alfa). The CHMP unanimously recommended full approval of Myozyme for the treatment of Pompe disease. Marketing authorization by the European Commission is expected within two to three months.
Upon approval, Myozyme will be the first treatment for Pompe disease and one of the first for an inherited muscle disorder. Pompe is a debilitating, progressive and often fatal disease affecting fewer than 10,000 people worldwide.
The marketing application for Myozyme included clinical data primarily from studies involving patients with infantile-onset Pompe disease. Genzyme initially focused its clinical development program on this population given the urgent medical need. In addition, the company believed it could more rapidly establish Myozyme's efficacy in this population and thereby make the product available sooner to all patients with Pompe disease. Genzyme recently initiated a clinical trial evaluating the safety and efficacy of Myozyme in patients with late-onset Pompe disease. This trial is designed to provide additional support for Myozyme's use. Results are expected to be available next year and will be submitted to regulatory authorities.
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