Asfotase alfa regulatory update

Alexion said Japan's Ministry of Health, Labor and Welfare (MHLW) granted Orphan Drug designation to asfotase alfa to treat hypophosphatasia (HPP), an inherited metabolic disease characterized by defective bone mineralization. The product, a fusion protein incorporating the catalytic domain of human tissue non-specific alkaline phosphatase ( TNSALP; ALPL) and a bone-targeting peptide, is under accelerated assessment in the EU (see BioCentury, July 28). Alexion began a rolling BLA to FDA for asfotase alfa in April. ...