Marisa Wexler MS,  —

Up to 1 in 4 people with paroxysmal nocturnal hemoglobinuria (PNH) who are treated with Ultomiris (ravulizumab-cwvz) or Soliris (eculizumab) experience clinically significant extravascular hemolysis — the destruction of red blood cells — but this isn’t typically associated with severe fatigue, a new global study reported. Soliris…

Treatment with Fabhalta (iptacopan) can ease fatigue and improve quality of life for people with paroxysmal nocturnal hemoglobinuria (PNH), according to data from two Phase 3 clinical trials. “These results … indicate that [Fabhalta] results in clinically meaningful disease control and meaningful improvements in the fatigue and [health-related…

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…

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,…

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…

Health Canada has approved the oral therapy Voydeya (danicopan) as an add-on to Ultomiris (ravulizumab) or Soliris (eculizumab) in adults with paroxysmal nocturnal hemoglobinuria (PNH) who have residual anemia due to blood cell destruction taking place outside blood vessels. The announcement came just a few months…

The U.S. Food and Drug Administration (FDA) has approved crovalimab — which works to reduce the destruction of red blood cells in paroxysmal nocturnal hemoglobinuria (PNH) — to treat teenagers and adults with PNH. The therapy, which won formal approval from the FDA on June 20, is…

Soliris (eculizumab) was successfully used to help manage paroxysmal nocturnal hemoglobinuria (PNH) during a pregnancy in two women. Based on observations made in these cases, the scientists noted that Soliris “seems to be generally safe in pregnancy for both mother and fetus,” though they emphasized a need for additional…

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…