An independent appraisal committee voted that there was adequate evidence to show that adding danicopan to a C5 inhibitor may benefit certain people with paroxysmal nocturnal hemoglobinuria (PNH), pending price listing. Committee members also decided the evidence isn’t adequate to show a net health benefit for Fabhalta (iptacopan)…
People in Japan using C5 inhibitors to treat their paroxysmal nocturnal hemoglobinuria (PNH) continue to experience symptoms like fatigue and shortness of breath, even if their hemoglobin levels surpass the median of real-world PNH populations, a survey study reports. “This study contributes to understanding the unmet needs of the…
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…
Almost 6 out of 10 people with paroxysmal nocturnal hemoglobinuria (PNH) living in the U.S. may be being given a higher initial loading dose of Ultomiris (ravulizumab) than is recommended on the medication’s label, a database study into its clinical use reports. Researchers also observed that maintenance doses,…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ruxoprubart, an experimental treatment for paroxysmal nocturnal hemoglobinuria (PNH) that’s now in Phase 2 clinical testing. The drug is designed to control the genetic disease while posing a lower infection risk than currently available treatments. Orphan…
The under-the-skin (subcutaneous) injection therapy crovalimab has been approved in China for adults and adolescents, ages 12 and older, with paroxysmal nocturnal hemoglobinuria (PNH) who have not previously received complement inhibitor medications. Crovalimab is expected to make treatment more convenient for PNH patients, given that it may be self-administered…
Fabhalta (iptacopan) and danicopan are two first-in-class treatments for paroxysmal nocturnal hemoglobinuria (PNH) that provide important health benefits in clinical trials, but uncertainties remain regarding their long-term efficacy and safety. That’s according to an evidence report from the Institute for…
NM8074, an investigational antibody therapy for paroxysmal nocturnal hemoglobinuria (PNH), will now be called ruxoprubart, its developer NovelMed has announced. The name change came after the USAN, or United States Adopted Names Council, selected ruxoprubart as the unique generic, or nonproprietary, name for the therapy candidate. According to…
Danicopan has been approved in Japan as a first-in-class, add-on therapy for people with paroxysmal nocturnal hemoglobinuria (PNH) who have an insufficient response to treatment with C5 inhibitors. Its developer Alexion, AstraZeneca Rare Disease will be marketing it under the brand name Voydeya, the company announced in a…
Danicopan, an experimental oral medication, continued to help control anemia when taken for up to 48 weeks by adults with paroxysmal nocturnal hemoglobinuria (PNH) who experience a breakdown of red blood cells outside of blood vessels — called extravascular hemolysis — despite being on a C5 inhibitor. That’s according…
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