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1 month ago | 
pnhnews.com | Andrea Lobo |Erin Fortin |Lindsey Shapiro |Marisa Wexler MS
An unusual meningococcal infection during treatment with Empaveli (pegcetacoplan) was reported in a 16-year-old girl with paroxysmal nocturnal hemoglobinuria (PNH), according to a case report from Finland. Meningococcal infections are a known risk for patients receiving complement inhibitors, such as Empaveli. As such, patients typically are vaccinated before starting treatment to prevent such infections.
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Jan 6, 2025 | 
pnhnews.com | Margarida Maia |Marisa Wexler MS |Erin Fortin |Steve Bryson
A novel combination of pozelimab and cemdisiran appears to be better than Ultomiris (ravulizumab) at controlling intravascular hemolysis — the rupture of red blood cells inside blood vessels — in adults with paroxysmal nocturnal hemoglobinuria (PNH), showing promise as a potential treatment for the rare acquired disease.
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Oct 14, 2024 | 
pnhnews.com | Andrea Lobo |Patricia Inacio |Erin Fortin |Margarida Maia
Treatment with Empaveli (pegcetacoplan), approved for adults with paroxysmal nocturnal hemoglobinuria (PNH), may lead to clinically meaningful improvements in blood parameters and fatigue among those with impaired bone marrow function. That’s according to a new analysis of data from two Phase 3 clinical trials — PEGASUS (NCT03500549) and PRINCE (NCT04085601) — that assessed the safety and efficacy of Empaveli in people with PNH.
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Oct 7, 2024 | 
pnhnews.com | Patricia Inacio |Brandi Lewis |Erin Fortin
In rare cases, acute kidney injury may be sign of paroxysmal nocturnal hemoglobinuria (PNH), as described in a recent case report. “Although renal dysfunction in PNH is rare and as renal dysfunction is a dreadful complication, timely intervention is life-saving for the patient who requires hemodialysis,” the scientists wrote. The report, “Paroxysmal nocturnal hemoglobinuria presenting as acute renal injury,” was published in the International Journal of Research in Medical Sciences.
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Sep 9, 2024 | 
pnhnews.com | Margarida Maia |Erin Fortin |Marisa Wexler MS |Andrea Lobo
The European Commission has approved Roche’s PiaSky (crovalimab) to treat adults and adolescents with paroxysmal nocturnal hemoglobinuria (PNH), ages 12 and older who weigh at least 40 kilograms (88 pounds). PiaSky is the first treatment for PNH in the European Union that’s administered subcutaneously (under the skin) once a month, and patients can be trained to administer it themselves. The therapy will likely be more convenient than other, similar treatments, which require regular infusions.
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Jul 8, 2024 | 
pnhnews.com | Margarida Maia |Erin Fortin |Lindsey Shapiro |Marisa Wexler MS
Most of the adults with paroxysmal nocturnal hemoglobinuria (PNH) who switched from Soliris (eculizumab) to PiaSky (crovalimab) in a clinical trial said they preferred PiaSky because it was easier to use, required fewer hospital visits, and took less time to administer.
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Jun 24, 2024 | 
pnhnews.com | Marisa Wexler MS |Erin Fortin |Patricia Inacio |Andrea Lobo
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 studies.
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Jun 17, 2024 | 
pnhnews.com | Patricia Inacio |Andrea Lobo |Erin Fortin
Epysqli (SB12), a biosimilar of Soliris (eculizumab), is as effective as the reference therapy in reducing the need for blood transfusions in patients with paroxysmal nocturnal hemoglobinuria (PNH). That’s according to a post-hoc analysis of the pivotal Phase 3 SB12 study (NCT04058158), whose findings supported Epysqli’s approval in the European Union (EU) following a positive recommendation from the EU’s Committee for Medicinal Products for Human Use (CHMP).
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May 6, 2024 | 
pnhnews.com | Andrea Lobo |Erin Fortin |Brandi Lewis |Margarida Maia
Dawn Health, a Danish digital health company, and Novartis have launched a smartphone app designed to help people living with paroxysmal nocturnal hemoglobinuria (PNH) with disease management. The free Ekiva PNH app helps patients track their symptoms and prepare for medical appointments, while also providing a content library where they can learn more about PNH and have “more informed and data-based conversations” with healthcare professionals, Dawn Health said.
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Apr 29, 2024 | 
pnhnews.com | Marisa Wexler MS |Erin Fortin |Steve Bryson
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 — that is, low counts of red blood cells because those cells are being destroyed in a process also known as hemolysis. Voydeya is given as an add-on to the standard-of-care therapies Ultomiris (ravulizumab) and Soliris (eculizumab).
