WASHINGTON (dpa-AFX) - Alexion Pharmaceuticals Inc. (ALXN) on Wednesday said it has filed a New Drug Application or NDA to Japan's Ministry of Health, Labour and Welfare or MHLW, for asfotase alfa, an investigational, first-in-class targeted enzyme replacement therapy for the treatment of hypophosphatasia or HPP.
HPP, a genetic metabolic disease, can lead to deformity of bones, profound muscle weakness, seizures, respiratory failure and cause premature death. The application was based on positive data from 71 patients with HPP including Japanese patients, enrolled in three pivotal prospective studies and their extensions, as well as a retrospective natural history study in infants.
Earlier in September, asfotase alfa was granted orphan drug designation by the MHLW for the treatment of patients with HPP. As a result, the NDA for asfotase alfa will receive priority review for marketing authorization, and, if approved, it would have 10 years of market exclusivity for the designated indication.
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