News Feature | October 21, 2014

Alexion Files NDA In Japan For Asfotase Alfa For HPP

By Estel Grace Masangkay

Alexion Pharmaceuticals announced that it has filed the New Drug Application (NDA) with the Ministry of Health, Labor, and Welfare (MHLW) in Japan for its asfotase alfa as treatment for hypophosphatasia (HPP).

Asfotase alfa is an investigational first-in-class targeted enzyme replacement therapy designed to correct the genetically defective metabolic process in hypophosphatasia. Through its mechanism of action, asfotase alfa prevents or reverses the complications arising from a life-long dysregulated mineral metabolism that characterizes the disease. Alexion acquired asfotase alfa along with its developer Enobia Pharma in 2011. The company received Breakthrough Therapy status from the U.S. Food and Drug Administration (FDA) for asfotase alfa last year in May.

The submission to the MHLW is supported by positive data from three pivotal prospective studies involving 71 HPP patients, including Japanese individuals. Leonard Bell, Chairman and CEO of Alexion, said, “The NDA submission for asfotase alfa is a critical step toward bringing this highly innovative and much-needed potential treatment to patients in Japan suffering from HPP. If approved, asfotase alfa would be the first therapy for patients with this devastating and life-threatening disorder.”

Hypophosphatasia is a genetic, chronic ultra-rare metabolic disease that results in the destruction and deformity of bones, extreme muscle weakness, respiratory failure, and death in patients of all ages. The disease is caused by mutations in the gene encoding an enzyme known as tissue non-specific alkaline phosphatase (TNSALP).

The MHLW granted Orphan Drug Designation to asfotase alfa last month, making it eligible for priority review, as well as 10 years of market exclusivity upon approval. The company has also filed a Biologics License Application (BLA) with the FDA for the same indication. Alexion reported in July that its Marketing Authorization Application (MAA) has been accepted by the European Medicines Agency (EMA).