Marisa Wexler MS,  —

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…

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…