News

Dawn Health, a Danish digital health company, and Novartis have launched a smartphone app designed to help people living with paroxysmal nocturnal hemoglobinuria (PNH) with disease management. The free Ekiva PNH app helps patients track their symptoms and prepare for medical appointments, while also providing a…

The oral therapy Voydeya (danicopan) is now approved in the European Union as an add-on treatment for paroxysmal nocturnal hemoglobinuria (PNH). The European Commission authorized the therapy for adults with PNH who have residual hemolytic anemia — that is, low counts of red blood cells because those…

The U.S. Food and Drug Administration (FDA) has given orphan drug designation to NM5072, an experimental treatment for anemia in people with paroxysmal nocturnal hemoglobinuria (PNH). The potential therapy showed safety and tolerability in an initial clinical trial in healthy volunteers, and a Phase 2 trial in PNH patients…

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.

Treatment with Soliris (eculizumab) or Ultomiris (ravulizumab) can substantially reduce the likelihood of death as a direct result of paroxysmal nocturnal hemoglobinuria (PNH), a new study found. While the findings showed that PNH itself is rarely fatal for people taking these therapies, co-occurring bone marrow failure remains…

The U.S. Food and Drug Administration (FDA) has approved Voydeya (danicopan) as an add-on treatment for extravascular hemolysis, or the destruction of red blood cells taking place outside blood vessels, in adults with paroxysmal nocturnal hemoglobinuria (PNH). The treatment is to be taken with the standard-of-care therapies Ultomiris…

Individualized dosing of Empaveli (pegcetacoplan) may allow for a larger interval between doses in about 30% of adults with paroxysmal nocturnal hemoglobinuria (PNH), while cutting costs by about 8%, according to a study that simulated a clinical trial. The study, “Development of a target concentration intervention to…

A European Medicines Agency committee has recommended that Novartis’ twice-daily oral therapy Fabhalta (iptacopan) be approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). The recommendation from the Committee for Medicinal Products for Human Use (CHMP) supports Fabhalta’s use as a monotherapy, or stand-alone treatment, in adults with…

An independent appraisal committee voted that there was adequate evidence to show that adding danicopan to a C5 inhibitor may benefit certain people with paroxysmal nocturnal hemoglobinuria (PNH), pending price listing. Committee members also decided the evidence isn’t adequate to show a net health benefit for Fabhalta (iptacopan)…

People in Japan using C5 inhibitors to treat their paroxysmal nocturnal hemoglobinuria (PNH) continue to experience symptoms like fatigue and shortness of breath, even if their hemoglobin levels surpass the median of real-world PNH populations, a survey study reports. “This study contributes to understanding the unmet needs of the…