News

Direct oral anticoagulants prevent recurring blood clotting in PNH

Direct oral anticoagulants, or DOACs, are safe and effective for preventing recurrent venous thromboembolism (VTE), or blood clots forming in a vein, in people with paroxysmal nocturnal hemoglobinuria (PNH), a study reports. These medications, given after standard vitamin K antagonists (VKAs) and low molecular weight heparin (LMWH) during the…

Case: Empaveli effectively treats extravascular hemolysis in girl, 14

A teenage girl with paroxysmal nocturnal hemoglobinuria (PNH) who experienced extravascular hemolysis, which occurs when red blood cells are destroyed outside of the blood vessels, saw her condition significantly improve after starting on Empaveli (pegcetacoplan) — a treatment currently approved only for adults with PNH. That’s according to…

As number of PNH cases rise in Italy, so does treatment cost: Study

Paroxysmal nocturnal hemoglobinuria (PNH) is being diagnosed more frequently in Italy, and while treatment with complement inhibitors can be effective, it carries a high yearly cost of over €133,000 (about $155,000) per patient, mostly due to medication expenses. According to a study, every fourth patient received treatment…

Fabhalta raises hemoglobin, ends transfusion need in PNH patients

Six months of Fabhalta (iptacopan) raised hemoglobin levels, eliminated the need for blood transfusions, and eased fatigue in most adults with paroxysmal nocturnal hemoglobinuria (PNH) who switched from standard anti-C5 therapies. That’s according to data from the open-label Phase 3b APPULSE-PNH (NCT05630001) that evaluated the therapy’s efficacy and…

Fabhalta boosts hemoglobin in PNH, eliminates need for transfusions

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…

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…