MHLW approves Alexion's new drug for the treatment of adult patients with PNH

recently, Alexion announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has approved Ultomiris (ravulizumab) for the treatment of adult patients with hairy sleep haemoglobinuria (PNH).U.S., Ultomiris was approved by the FDA in December 2018 for the treatment of adult patients with PNH. Currently, Ultomiris' applications for the treatment of adult patients with PNH are also under review by EU regulators. In the United States, Japan and the European Union, Ultomiris has been granted the status of an orphan drug for the treatment of PNH.Ultomirisis a long-acting C5 supplement inhibitor that suppresses C5 proteins in the body's immune system's cascading response. The drug has been positioned as an upgraded version of Alexion's heavyweight drug Soliris, which was first approved for market in 2007 and has been approved for treatment of three rare diseases: PNH, atypical hemolytic uremia syndrome (aHUS), anti-acetylcholine lipolybo-positive systemic severe muscle weakness (gMG).addition, Soliris' treatment of spinal corditis-optic neurospectral disorder (NMOSD) entered FDA priority review in U.S. regulation at the end of February.Relevant StudiesThis approval is based on comprehensive data from two key Phase III clinical studies, the largest Phase III study ever conducted in PNH patients, with 441 patients in the group, including those who had never been treated with complementary inhibitors, and those who received a stable PNH condition from soliris (eculizumab).data show that Ultomiris infusions every eight weeks and Soliris infusions every 2 weeks are non-poor in terms of all 11 end points (major end points and all key secondary endpoints).study, the security of Ultomiris is similar to that of Soliris. Additional data recently released show that Ultomiris provides immediate and complete C5 inhibition for up to eight weeks, while eliminating the breakthrough hemolysis associated with incomplete C5 inhibition.
(Drug Information Network)