Alexion Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Company’s Biologics License Application (BLA) for asfotase alfa, an investigational, first-in-class enzyme replacement therapy for treatment of patients with infantile- and juvenile-onset hypophosphatasia (HPP). The BLA submission is supported by data from 71 treated patients with HPP enrolled in three prospective studies and their extensions, as well as two retrospective natural history studies.

“If approved, asfotase alfa will be the first treatment for patients with HPP, a devastating disease that can result in impaired respiratory function, severe disability and premature death for some patients,” said Leonard Bell, M.D., Chairman and Chief Executive Officer of Alexion. “The FDA's acceptance of our BLA for Priority Review is a significant step toward bringing this highly innovative and much-needed potential treatment to patients in the United States suffering from HPP.”

In May 2013, the FDA granted Breakthrough Therapy designation for asfotase alfa and in April 2014, Alexion initiated the rolling submission of the BLA. Breakthrough Therapy designation is designed to expedite the development of a drug to treat a serious or life-threatening disease when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. Priority Review designation is given to drugs that treat a serious condition and, if approved, would provide a significant improvement in safety or effectiveness. Alexion has also submitted a Marketing Authorization Application for asfotase alfa with the European Medicines Agency and has submitted a New Drug Application for asfotase alfa to Japan’s Ministry of Health, Labour and Welfare.

For more details, go to: http://news.alexionpharma.com/press-release/company-news/fda-grants-priority-review-asfotase-alfa-treatment-patients-hypophosphata