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.
News
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…
A European Medicines Agency committee has recommended the approval of Voydeya (danicopan) as an add-on treatment for adults with paroxysmal nocturnal hemoglobinuria (PNH) who continue to experience anemia despite treatment with C5 inhibitors. Regulators will use this guidance from the Committee for Medicinal Products for Human Use (CHMP) when…
Almost 6 out of 10 people with paroxysmal nocturnal hemoglobinuria (PNH) living in the U.S. may be being given a higher initial loading dose of Ultomiris (ravulizumab) than is recommended on the medication’s label, a database study into its clinical use reports. Researchers also observed that maintenance doses,…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ruxoprubart, an experimental treatment for paroxysmal nocturnal hemoglobinuria (PNH) that’s now in Phase 2 clinical testing. The drug is designed to control the genetic disease while posing a lower infection risk than currently available treatments. Orphan…
Recent Posts
- Rethinking what control looks like after a PNH diagnosis
- New study backs approved Voydeya dosing for PNH add-on treatment
- In the rare disease community, we find strength in each other
- Complement biomarkers may not reliably track PNH disease activity
- Improving quality of life is key to living fully with PNH