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…
News
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…
In rare cases, acute kidney injury may be sign of paroxysmal nocturnal hemoglobinuria (PNH), as described in a recent case report. “Although renal dysfunction in PNH is rare and as renal dysfunction is a dreadful complication, timely intervention is life-saving for the patient who requires hemodialysis,” the scientists wrote.
Treatment with Ultomiris (ravulizumab) enabled paroxysmal nocturnal hemoglobinuria (PNH) patients to achieve the same level of physical and mental health, daily activity, and sleep duration displayed by healthy individuals from the U.S. general population, according to the findings of an observational study. In the study, people with PNH…
Recent Posts
- Biogen to acquire Apellis, adding PNH treatment Empaveli to portfolio
- 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