Marisa Wexler MS,  —

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…

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…

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…

Danicopan has been approved in Japan as a first-in-class, add-on therapy for people with paroxysmal nocturnal hemoglobinuria (PNH) who have an insufficient response to treatment with C5 inhibitors. Its developer Alexion, AstraZeneca Rare Disease will be marketing it under the brand name Voydeya, the company announced in a…

NovelMed Therapeutics has launched a Phase 2 clinical trial to test its experimental therapy, NM8074, in people with paroxysmal nocturnal hemoglobinuria (PNH) who haven’t received treatment for the disease. Eight patients have been enrolled so far and two have started treatment, according to the company. The Phase…

A proof-of-concept clinical trial of the experimental oral therapy BCX10013 is enrolling people with paroxysmal nocturnal hemoglobinuria (PNH) in Cape Town, South Africa, with plans to expand to other sites where approved PNH treatments are not available. The Phase 1 study (NCT06100900) is open to adults with PNH…

The experimental treatment cemdisiran (ALN-CC5) failed to fully control hemolysis, or red blood cell destruction, in a small clinical trial involving people with paroxysmal nocturnal hemoglobinuria (PNH). However, the therapy’s use was found to reduce the dose of Soliris (eculizumab) needed to keep blood cell destruction under control…

The U.S. Food and Drug Administration (FDA) has agreed to review an application seeking the approval of the monthly subcutaneous (under-the-skin) injection therapy crovalimab as a treatment for paroxysmal nocturnal hemoglobinuria (PNH), according to its developer, Genentech. “This filing acceptance reinforces the value of crovalimab,” Levi Garraway, MD,…

When used in real-world practice to treat paroxysmal nocturnal hemoglobinuria (PNH), the safety and effectiveness of Ultomiris (ravulizumab-cwvz) is similar to what’s been reported in clinical trials, according to a recent study from Japan. “These real-world data support the safety and effectiveness of [Ultomiris] treatment in Japanese patients,”…