Alexion's Asfotase Alfa For HPP Receives Orphan Status In Japan
Alexion Pharmaceuticals reported that it has received Orphan Drug Designation from the Japanese Ministry of Health, Labor, and Welfare (MHLW) for its asfotase alfa as treatment for patients with hypophosphatasia (HPP).
Asfotase alfa is an investigational first-in-class targeted enzyme replacement therapy developed for HPP. The treatment targets the root of the disease and normalizes the genetically defective metabolic process that causes it. Through its mechanism of action, asfotase alfa prevents or reverses the serious complications of life-long dysregulated mineral metabolism that characterizes HPP.
Hypophosphatasia is a genetic, chronic, and progressive ultra-rare metabolic disease. HPP causes defective bone mineralization and can result in deformed bones, severe muscle weakness, respiratory failure, seizures, and premature death.
“HPP is a devastating disease for patients and families and there are currently no approved treatment options. The orphan drug designation granted for asfotase alfa underscores the significant need for an effective treatment option for Japanese patients suffering from this severe, ultra-rare disorder,” said Dr. Martin Mackay, EVP and Global Head of R&D at Alexion.
The MHLW grants orphan status to drugs addressing serious diseases that affect less than 50,000 in Japan. Orphan status provides priority review for marketing authorization as well as a period of 10 years of marketing exclusivity upon approval of the drug for the designated indication.
The drug received Breakthrough Therapy designation last year from the U.S. Food and Drug Administration (FDA). Alexion has also filed a rolling Biologics License Application (BLA) submission for the drug in April. The company also announced the recent acceptance for accelerated assessment of its Marketing Authorization Application (MAA) for asfotase alfa in the EU.
Earlier this month, Alexion won the backing of U.K.’s National Institute for Health and Care Excellence (NICE) for its drug Soliris (eculizumab) as treatment for atypical Haemolytic Uraemic Syndrome (aHUS).