Steve Bryson PhD,  —

One year of treatment with Fabhalta (iptacopan) boosts hemoglobin levels, eliminates the need for blood transfusions, and eases fatigue in most adults with paroxysmal nocturnal hemoglobinuria (PNH). That’s according to final data from two global Phase 3 clinical trials: APPLY-PNH (NCT04558918) and APPOINT-PNH (NCT04820530), which evaluated…

Novelmed Therapeutics’ ruxoprubart prevented red blood cell destruction in treatment-naïve adults with paroxysmal nocturnal hemoglobinuria (PNH), allowing these untreated patients to avoid blood transfusions altogether. That’s according to 12-week interim data from an ongoing Phase 2 clinical trial (NCT05646524), which showed that the PNH treatment, when administered…

Epysqli (eculizumab-aagh), a Soliris (eculizumab) biosimilar, is now available in the U.S. to reduce red blood cell destruction (hemolysis) in people with paroxysmal nocturnal hemoglobinuria (PNH). A biosimilar contains an active ingredient that’s highly similar to an approved biological medicine, or reference product, with no clinically meaningful differences…

Samsung Bioepis has partnered with Teva Pharmaceuticals to commercialize the Soliris (eculizumab) biosimilar Epysqli (eculizumab-aagh) in the U.S. for people with paroxysmal nocturnal hemoglobinuria (PNH). A biosimilar is a biological product with an active ingredient that is highly similar to an already approved biological medicine, known as…

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…