A tablet has been approved for a common complication during treatment for a rare blood disorder.
The US Food and Drug Administration has approved danicopan as an add-on therapy to treat extravascular hemolysis in adults receiving ravulizumab orPNH is a rare blood disorder affecting 1-10 individuals per million. The condition, which eliminates red blood cells and leads to blood clots and impaired bone marrow function, can cause life-threateningRavulizumab and eculizumab, also both made by AstraZeneca, inhibit the destruction of red blood cells.
Danicopan, an investigational, first-in-class, oral complement factor D inhibitor, is designed to control intravascular hemolysis and prevent extravascular hemolysis.in 63 patients with PNH who received ravulizumab or eculizumab and experienced significant extravascular hemolysis. These patients were randomized 2:1 to either danicopan or placebo.
Danicopan add-on significantly improved hemoglobin concentrations at 12 weeks and made transfusions less likely.M. Alexander Otto is a physician assistant with a master's degree in medical science and a journalism degree from Newhouse. He is an award-winning medical journalist who worked for several major news outlets before joiningAll material on this website is protected by copyright, Copyright © 1994-2024 by WebMD LLC. This website also contains material copyrighted by 3rd parties.
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