News

Taking Novartis’ Fabhalta (iptacopan) as a monotherapy — meaning as a sole treatment — twice daily for almost one year continued to sustain hemoglobin at near-normal levels, generally without the need for blood transfusions, in adults with paroxysmal nocturnal hemoglobinuria (PNH). These new data come from an open-label…

Treatment with Empaveli (pegcetacoplan) — a C3 protein blocker — effectively improved disease control and health-related quality of life, while reducing fatigue in people with paroxysmal nocturnal hemoglobinuria (PNH) in the U.S. and Europe, a real-world study reported. Study findings also showed higher physician and patient satisfaction with…

The U.S. Food and Drug Administration (FDA) has approved iptacopan for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). It will be sold under the brand name Fabhalta. As the first oral monotherapy approved for the rare condition in the U.S., Fabhalta is expected to become available…

Add-on danicopan treatment led to better control of anemia among paroxysmal nocturnal hemoglobinuria (PNH) patients experiencing residual red blood cell destruction, or hemolysis, while on Soliris (eculizumab) or Ultomiris (ravulizumab), according to data from a Phase 3 clinical trial. The addition of the investigational oral therapy to…

A rare case of portal vein thrombosis (PVT) — where a clot blocks blood flow in the portal vein, a major liver blood vessel — in a 49-year-old woman with paroxysmal nocturnal hemoglobinuria (PNH) was detailed in a case report. Transjugular intrahepatic portosystemic shunt (TIPS), a surgical procedure that…

Early mortality in people with paroxysmal nocturnal hemoglobinuria (PNH) is linked to being older and having heart or blood disorders and a history of solid tumors at diagnosis, a Danish study has found. The leading causes of death among patients were infections and associated blood-related diseases, while the approved…

An enzyme called lactate dehydrogenase (LDH), which is a marker of red blood cell breakdown, may be used as a proxy measure for fatigue and quality of life in people with paroxysmal nocturnal hemoglobinuria (PNH) who are being treated with Ultomiris (ravulizumab) or Soliris (eculizumab). That’s according…

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…

Paroxysmal nocturnal hemoglobinuria (PNH) developed in a young man after an asymptomatic infection with SARS-CoV-2, the virus that causes COVID-19, a recent case study reports. The study, “Paroxysmal nocturnal hemoglobinuria in a patient post COVID-19 virus infection: A case report with literature review,” was published in…