FDA Accepts Alexion’s BLA for Asfotase AlfaBy
Alexion Pharmaceuticals, a biopharmaceutical company specializing in developing drugs for rare diseases, reports that the US 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).
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.
Source: Alexion Pharmaceuticals