News

Ruxoprubart treatment for PNH found effective in trial: Data

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…

Soliris may boost pregnancy outcomes for women with PNH

Pregnant women with paroxysmal nocturnal hemoglobinuria (PNH) who receive Soliris (eculizumab) have higher chances of their babies surviving compared with those who don’t, with fewer miscarriages before 24 weeks, a meta-analysis study has found. Biosimilars of Soliris are also available. “Eculizumab appears to be safe and benefits pregnant women with…

Extravascular hemolysis affects PNH patients on Soliris, Ultomiris

Up to 1 in 4 people with paroxysmal nocturnal hemoglobinuria (PNH) who are treated with Ultomiris (ravulizumab-cwvz) or Soliris (eculizumab) experience clinically significant extravascular hemolysis — the destruction of red blood cells — but this isn’t typically associated with severe fatigue, a new global study reported. Soliris…

In Phase 3 studies, Fabhalta eases fatigue, improves life quality in PNH

Treatment with Fabhalta (iptacopan) can ease fatigue and improve quality of life for people with paroxysmal nocturnal hemoglobinuria (PNH), according to data from two Phase 3 clinical trials. “These results … indicate that [Fabhalta] results in clinically meaningful disease control and meaningful improvements in the fatigue and [health-related…

Epysqli, biosimilar of PNH treatment Soliris, available in US

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…

Empaveli dose adjustments thwart hemolysis in patient’s surgeries

Close monitoring and Empaveli (pegcetacoplan) dose adjustments before and after surgery effectively prevented episodes of breakthrough hemolysis,in a 67-year-old man with paroxysmal nocturnal hemoglobinuria (PNH), according to a case report in Spain. Hemolysis is a hallmark symptom of PNH marked by the breakdown of red blood cells, which…

Epysqli safety and efficacy similar in Asian, non-Asian PNH patients

Epysqli (SB12), a biosimilar of Soliris (eculizumab), is comparable in safety and efficacy for Asian and non-Asian patients with paroxysmal nocturnal hemoglobinuria (PNH). That’s according to a post-hoc analysis of the pivotal Phase 3 SB12 study (NCT04058158), the findings of which supported Epysqli’s approval in the U.S.

Omeros launches Phase 3 program of zaltenibart as PNH treatment

Clinical site activation is now ongoing for a Phase 3 program that will evaluate zaltenibart (OMS906) as a treatment for people with paroxysmal nocturnal hemoglobinuria (PNH), its developer Omeros has announced. A total of 120 sites across 30 countries were chosen for clinical trial participation, according to a…