News

People with paroxysmal nocturnal hemoglobinuria (PNH) who switch to Empaveli (pegcetacoplan) after having an inadequate response to other, older treatments usually see good efficacy with the newer therapy, according to a study that looked at real-world data in Italy. Indeed, according to the researchers, “most patients exhibited a…

The use of Fabhalta (iptacopan) — approved in the U.S. in December 2023 as a treatment for paroxysmal nocturnal hemoglobinuria (PNH) — may save about $4 million over a patient’s lifetime compared with intravenous, or into-the-vein, standard of care therapies, according to a study by U.S. researchers. The oral…

Most Chinese adults and adolescents with paroxysmal nocturnal hemoglobinuria (PNH) no longer require blood transfusions after a median of six months of treatment with Soliris (eculizumab). That’s according to a small study at a single center in China, which also found the approved therapy to be safe and…

Health Canada has approved the oral therapy Fabhalta (iptacopan) as a treatment for adults with paroxysmal nocturnal hemoglobinuria (PNH) who have hemolytic anemia. With that decision, Fabhalta has now become the first oral medication that can be used on its own to control PNH to win approval in…

Long-term treatment with Ultomiris (ravulizumab) reduced the risk of death by five times in people with paroxysmal nocturnal hemoglobinuria (PNH), and also tended to eliminate the need for blood transfusions and to improve patients’ quality of life, a new analysis suggests. The findings were detailed in the study,…

Voydeya (danicopan) effectively and safely treated paroxysmal nocturnal hemoglobinuria (PNH) in people with significant extravascular hemolysis, when used as an add-on therapy to Ultomiris (ravulizumab) or Soliris (eculizumab). That’s according to long-term data from the Phase 3 ALPHA trial (NCT04469465), which found that dual treatment with…

Fatigue affects the ability of adults with paroxysmal nocturnal hemoglobinuria (PNH) to perform daily tasks, interact socially, and stay focused, even with treatment, showing how the disease limits physical, emotional, and social well-being, according to a real-world study. The findings indicate how the impact on health-related quality of life…

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…

A novel combination of pozelimab and cemdisiran appears to be better than Ultomiris (ravulizumab) at controlling intravascular hemolysis — the rupture of red blood cells inside blood vessels — in adults with paroxysmal nocturnal hemoglobinuria (PNH), showing promise as a potential treatment for the rare acquired disease. That’s…

Patients with paroxysmal nocturnal hemoglobinuria (PNH) in 53 countries with limited access to life-saving treatments will be able to receive treatment with  Fabhalta (iptacopan). The Max Foundation, a global nonprofit organization, and Novartis are expanding their collaboration to address the unmet needs of patients across Sub-Saharan…