News

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…

This year, PNH News brought up-to-date coverage of the latest scientific and clinical research developments in paroxysmal nocturnal hemoglobinuria (PNH). Here are the 10 most-read news stories of 2023, along with a brief description of what made them interesting to our readers. We look forward to continuing to…

Long-term treatment with Soliris (eculizumab) safely and effectively reduced the severity of paroxysmal nocturnal hemoglobinuria (PNH) in Korean adults with high disease activity, according to a real-world study. Soliris was found to ease PNH symptoms, including fatigue, anemia — low levels of hemoglobin, the protein in red…

Taking Novartis’ Fabhalta (iptacopan) as a monotherapy — meaning as a sole treatment — twice daily for almost one year continued to sustain hemoglobin at near-normal levels, generally without the need for blood transfusions, in adults with paroxysmal nocturnal hemoglobinuria (PNH). These new data come from an open-label…

Treatment with Empaveli (pegcetacoplan) — a C3 protein blocker — effectively improved disease control and health-related quality of life, while reducing fatigue in people with paroxysmal nocturnal hemoglobinuria (PNH) in the U.S. and Europe, a real-world study reported. Study findings also showed higher physician and patient satisfaction with…