News

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…

Soliris (eculizumab) treatment was associated with a nearly 50% reduction in mortality risk and a 60% lower risk of blood clots or other major blood vessel-related events in people with paroxysmal nocturnal hemoglobinuria (PNH), according to data from a global registry study. Those having the greatest survival benefits…

The U.S. Food and Drug Administration (FDA) has cleared for use an injector device intended to make self-administration of Empaveli (pegcetacoplan) more convenient for adults with paroxysmal nocturnal hemoglobinuria (PNH). Empaveli, marketed as Aspaveli in Europe, is given via under-the-skin (subcutaneous) injections. Before the FDA decision, patients were…

People with paroxysmal nocturnal hemoglobinuria (PNH) who had a prior blood clot-related event are more likely to have such events again, regardless of whether they recently used blood thinners, a real-life registry study found. Other factors that appeared to increase their likelihood in PNH patients not on Soliris…

Long-term treatment with Empaveli (pegcetacoplan) was generally well tolerated among adults with paroxysmal nocturnal hemoglobinuria (PNH), with the rate of injection-site reactions declining over time. That’s according to data from the Phase 3 PEGASUS clinical trial (NCT03500549) and the open-label extension (OLE) Study 307 (NCT03531255) that spanned…

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,…

From signing up for a patient panel webinar to swapping out social media profile photos, participants in PNH Awareness Week — observed this year Sep. 3-10 — are doing their part to call attention to paroxysmal nocturnal hemoglobinuria (PNH), a very rare acquired disease with which some 500 U.S.

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,”…