Alexion Pharmaceuticals, Inc. (ALXN) said that Japan's Ministry of Health, Labour and Welfare or MHLW has granted orphan drug designation to asfotase alfa to treat patients with hypophosphatasia or HPP, a genetic, chronic and progressive ultra-rare metabolic disease characterized by defective bone mineralization. HPP patients can experience a range of devastating results like the destruction and deformity of bones, profound muscle weakness, seizures, respiratory failure and premature death.
Asfotase alfa is an investigational, highly innovative, first-in-class targeted enzyme replacement therapy designed to address the underlying cause of HPP.
Based on the opinion of the Pharmaceutical Affairs and Food Sanitation Council, the Ministry of Health, Labour and Welfare grants orphan status to drugs and medical devices that treat serious diseases of high unmet medical need that affect fewer than 50,000 patients in Japan. Orphan drug designation provides drug developers with certain benefits and incentives, including priority review for marketing authorization and a period of 10 years of market exclusivity if regulatory approval is received for the designated indication.
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Business News
May 15, 2026 15:25 ET Apart from the confirmation of Kevin Warsh as the next Fed chair, the main news on the economics front this week included key price data from the U.S. and the first quarter economic growth figures from major economies. Both consumer prices and producer costs have started to reflect the effect of supply shocks due to the Middle East conflict. In Europe, GDP data was in focus, while inflation data from China dominated the news flow in Asia.