First nmosd drug! New indications of the alexion complement C5 inhibitor soliris have been approved by Japan!

November 23, 2019 / BIOON / -- alexion is a biopharmaceutical company dedicated to the research and development of new drugs for rare diseases Recently, the company announced that Japan's Ministry of health, labor and welfare (MHLW) has approved a new indication of soliris (eculizumab), which is used to treat patients with nmosd (including optic neuropathic myelitis) with positive AQP4 antibody and prevent relapse This approval makes soliris the first and only drug approved to treat nmosd in Japan In the United States and the European Union, soliris was approved in June and August this year to treat patients with anti AQP4 antibody positive nmosd The drug is also the first drug approved in the United States and Europe to treat nmosd In the United States, the European Union and Japan, soliris has been granted orphan drug qualification (odd) to treat nmosd patients Nmosd is a rare, serious, destructive, complement mediated autoimmune disease of the central nervous system Attacking the central nervous system without warning can cause progressive and irreversible damage to the brain, optic nerve and spinal cord, which may lead to long-term disability The disease is characterized by relapses, each of which leads to progressive accumulation of disability and may lead to premature death Complement activation induced by anti-AQP4 antibody is one of the main potential causes of these damages About three quarters (73%) of nmosd patients are positive for anti-AQP4 autoantibody The detection of anti AQP4 autoantibodies can be used for the diagnosis of nmosd, which mainly affects women, usually in the golden age of their lives Soliris new indications approval based on data from phase III clinical study event The study was carried out in patients with nmosd positive for anti AQP4 autoantibodies and evaluated the efficacy and safety of soliris compared to placebo The results showed that the study reached the primary end point, and soliris significantly reduced the risk of recurrence of nmosd by 94.2% (P < 0.0001) compared with placebo At the 48th week of treatment, 97.9% of the patients in the soliris treatment group did not relapse, and 63.2% in the placebo group In addition, compared with placebo, soliris also significantly reduced the annual recurrence rate during the study period by 95.5% (P < 0.0001), reaching the key secondary end point of the study In this study, soliris was well tolerated and its safety was consistent with the previous study and the actual clinical practice of three approved indications in the real world Soliris is a pioneering complement inhibitor, which works by inhibiting C5 protein in the terminal part of complement cascade reaction Complement cascade reaction is a part of the immune system Its uncontrolled activation plays an important role in severe rare and super rare diseases, including paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), myasthenia gravis (mg) and nmosd with positive anti acetylcholine receptor (AChR) antibody Soliris was first approved for marketing in 2007 At present, it has approved four super rare diseases: PNH, aHUS, anti AChR antibody positive GMG and nmosd The drug is one of the world's best-selling orphanages, with sales of $3.563 billion in 2018, accounting for 86.3% of alexion's sales Analysts predict that nmosd indications will add about $700 million to soliris's sales In addition to expanding the indications of soliris, alexion is also developing the upgraded product ultomiris, which was approved for PNH indications by the United States, Japan and the European Union in December 2018, June 2019 and July 2019, respectively In October this year, the new indications of ultomiris for aHUS were also approved by FDA Ultomiris is the first and only long-acting C5 complement inhibitor given every 8 weeks In the phase III clinical study of PNH treatment, ultomiris infusion every 2 months (8 weeks) and soliris infusion every 2 weeks achieved non inferiority in all 11 endpoints The industry predicts that ultomiris will become a new standard for clinical treatment of PNH Based on the strong clinical data and differential characteristics, ultomiris will occupy the majority of PNH market share after going on the market, including PNH primary patients and treated PNH patients transferred from soliris to ultomiris The total sales of these two drugs in 2022 is expected to reach 5 billion US dollars Evaluatepharma, a pharmaceutical market research firm, predicts that ultomiris' sales will reach $3.43 billion in 2024 At present, alexion is actively promoting the market penetration of ultomiris, and is also actively developing other indications of ultomiris (including aHUS, GMG) and the development of hypodermic injection type ultomiris Origin of the original text: solidis (eculizumab) receives approval in Japan for the prevention of relapse in patients with neuroscience optica spectrum disorder (nmosd)