Lindsey Shapiro, PhD,  —

A European Medicines Agency committee has recommended the approval of Voydeya (danicopan) as an add-on treatment for adults with paroxysmal nocturnal hemoglobinuria (PNH) who continue to experience anemia despite treatment with C5 inhibitors. Regulators will use this guidance from the Committee for Medicinal Products for Human Use (CHMP) when…

The U.S. Food and Drug Administration (FDA) has approved iptacopan for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). It will be sold under the brand name Fabhalta. As the first oral monotherapy approved for the rare condition in the U.S., Fabhalta is expected to become available…

Add-on danicopan treatment led to better control of anemia among paroxysmal nocturnal hemoglobinuria (PNH) patients experiencing residual red blood cell destruction, or hemolysis, while on Soliris (eculizumab) or Ultomiris (ravulizumab), according to data from a Phase 3 clinical trial. The addition of the investigational oral therapy to…

Soliris (eculizumab) treatment was associated with a nearly 50% reduction in mortality risk and a 60% lower risk of blood clots or other major blood vessel-related events in people with paroxysmal nocturnal hemoglobinuria (PNH), according to data from a global registry study. Those having the greatest survival benefits…

The U.S. Food and Drug Administration (FDA) has cleared for use an injector device intended to make self-administration of Empaveli (pegcetacoplan) more convenient for adults with paroxysmal nocturnal hemoglobinuria (PNH). Empaveli, marketed as Aspaveli in Europe, is given via under-the-skin (subcutaneous) injections. Before the FDA decision, patients were…

Long-term treatment with Empaveli (pegcetacoplan) was generally well tolerated among adults with paroxysmal nocturnal hemoglobinuria (PNH), with the rate of injection-site reactions declining over time. That’s according to data from the Phase 3 PEGASUS clinical trial (NCT03500549) and the open-label extension (OLE) Study 307 (NCT03531255) that spanned…