Steve Bryson PhD,  —

Six months of twice-daily treatment with oral Fabhalta (iptacopan) improved hemoglobin levels in adults with paroxysmal nocturnal hemoglobinuria (PNH) who switched from standard anti-C5 therapies, according to top-line results from a Phase 3 clinical trial. Dubbed APPULSE-PNH (NCT05630001), the now-complete study tested Novartis’ therapy in 52…

Omeros’ zaltenibart (OMS906) as a single therapy has shown promise as a treatment for paroxysmal nocturnal hemoglobinuria (PNH) in a small clinical study, normalizing signs of hemolysis, or red blood cell destruction, in adults with hard-to-treat disease, according to new interim data. The trial enrolled PNH patients who…

England’s National Institute for Health and Care Excellence (NICE) has recommended that the oral treatment Voydeya (danicopan) be covered by the country’s National Health Service (NHS) and provided at low or no cost to certain adults with paroxysmal nocturnal hemoglobinuria (PNH). The recommendation covers Voydeya’s use as an…

Long-term treatment with KP104, a first-in-class dual-function complement inhibitor, controlled red blood cell destruction occurring both inside and outside the blood vessels of people with paroxysmal nocturnal hemoglobinuria (PNH), according to data from a Phase 2 trial. Based on these findings, Kira Pharmaceuticals, its developer,…

Onco360 will supply Voydeya (danicopan), an oral add-on medication for treating extravascular hemolysis (EVH), or red blood cell destruction occurring outside blood vessels, to adults with paroxysmal nocturnal hemoglobinuria (PNH). Alexion Pharmaceuticals selected the pharmacy, a subsidiary of BrightSpring Health Services, as a national pharmacy partner.

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…

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…

Paroxysmal nocturnal hemoglobinuria (PNH) developed in a young man after an asymptomatic infection with SARS-CoV-2, the virus that causes COVID-19, a recent case study reports. The study, “Paroxysmal nocturnal hemoglobinuria in a patient post COVID-19 virus infection: A case report with literature review,” was published in…

Treatment with OMS906 is showing an ability to normalize all markers of red blood cell destruction, or hemolysis, in severely anemic people with paroxysmal nocturnal hemoglobinuria (PNH) never given a complement inhibitor. According to a five-month interim analysis of a Phase 1b proof-of-concept study (NCT05889299), once monthly, under-the-skin…