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…
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…
Note: This story was updated Dec. 6, 2024, to correct that Voydeya is approved as an add-on treatment to standard PNH therapies to help control extravascular hemolysis. The headline was changed to clarify that these were the results of a review study. Voydeya (danicopan) — whether given as a…
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…
Treatment with Ultomiris (ravulizumab) successfully reduced multiple blood clots that didn’t respond to standard anti-clotting therapy in a woman with paroxysmal nocturnal hemoglobinuria (PNH), a case study shows. “This report presents a case of myocardial and renal infarction [heart and kidney tissue death due to blood supply obstruction]…
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…
The cases of three people with paroxysmal nocturnal hemoglobinuria (PNH) who experienced serious complications after switching from an experimental medicine to the approved PNH treatment Ultomiris (ravulizumab) highlight the potential for serious risks when switching from a PNH treatment targeting earlier steps of complement activation to one targeting…
Treatment with Empaveli (pegcetacoplan), approved for adults with paroxysmal nocturnal hemoglobinuria (PNH), may lead to clinically meaningful improvements in blood parameters and fatigue among those with impaired bone marrow function. That’s according to a new analysis of data from two Phase 3 clinical trials — PEGASUS (NCT03500549) and…
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