Health regulators in Japan have given Alexion Pharmaceuticals Inc. the green light to market its new drug Ultomiris for a second rare blood disease.
The drug, the company’s follow-up to its blockbuster Soliris, is already approved in that country for patients with paroxysmal nocturnal hemoglobinuria (PNH), also a rare blood disorder.
The company announced the latest decision by Japan’s Ministry of Health, Labour and Welfare on Friday. It allows Alexion to market the drug to adults and children with atypical hemolytic uremic syndrome (aHUS).
The ultra-rare disease can cause progressive injury to vital organs, primarily the kidneys, via blood clots and damage to the walls of the blood vessels.
Like Soliris, Ultomiris works by inhibiting C5, an immune system protein that is overactivated in people with the disease.
“Today’s approval is another important step in our efforts to continue innovating for patients and improving their treatment experience,” John Orloff, MD, who heads R&D for Alexion, said in a statement.
The drug requires fewer infusions than the current treatment, Soliris. It is given every other month for adults and children who are at least 44 pounds, and monthly for children under 44 pounds.
The company is looking to switch Soliris patients to the new drug, which it touts as having potential to be the new standard treatment for the disease in Japan.
Ultomiris is also approved to treat aHUS in the U.S. and in Europe, where the company is also close to gaining approval on a newer version of the drug.
Contact Natalie Missakian at news@newhavenbiz.com
