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1 month ago | 
pnhnews.com | Andrea Lobo |Margarida Maia |Marisa Wexler MS |Katherine Poinsatte
Clinical site activation is now ongoing for a Phase 3 program that will evaluate zaltenibart (OMS906) as a treatment for people with paroxysmal nocturnal hemoglobinuria (PNH), its developer Omeros has announced. A total of 120 sites across 30 countries were chosen for clinical trial participation, according to a company press release, which noted that investigators at “a good number of” these sites have already identified eligible and available patients to enroll.
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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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1 month ago | 
pnhnews.com | Marisa Wexler MS |Margarida Maia |Lindsey Shapiro |Andrea Lobo
People with paroxysmal nocturnal hemoglobinuria (PNH) who switch to Empaveli (pegcetacoplan) after having an inadequate response to other, older treatments usually see good efficacy with the newer therapy, according to a study that looked at real-world data in Italy. Indeed, according to the researchers, “most patients exhibited a complete or good response,” with less need for blood transfusions. None of the individuals who switched experience thrombosis, when a blood clot blocks a blood vessel.
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1 month ago | 
pnhnews.com | Margarida Maia |Brandi Lewis |Marisa Wexler MS |Steve Bryson
The use of Fabhalta (iptacopan) — approved in the U.S. in December 2023 as a treatment for paroxysmal nocturnal hemoglobinuria (PNH) — may save about $4 million over a patient’s lifetime compared with intravenous, or into-the-vein, standard of care therapies, according to a study by U.S. researchers. The oral therapy was found, in the clinical studies that served as the basis for its U.S. approval, to be more convenient for patients than the use of other complement inhibitors for treating PNH.
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2 months ago | 
pnhnews.com | Margarida Maia |Steve Bryson |Marisa Wexler MS |Brandi Lewis
Most Chinese adults and adolescents with paroxysmal nocturnal hemoglobinuria (PNH) no longer require blood transfusions after a median of six months of treatment with Soliris (eculizumab). That’s according to a small study at a single center in China, which also found the approved therapy to be safe and well tolerated.
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2 months ago | 
ankylosingspondylitisnews.com | Marisa Wexler MS |Esteban Cerezo |Andrea Lobo |Lindsey Shapiro
The U.S. Food and Drug Administration (FDA) has agreed to review an application seeking approval of AVT05, a proposed biosimilar for Simponi (golimumab), which is an approved treatment for ankylosing spondylitis. Developers Alvotech and Teva Pharmaceuticals said this marks the first time that the FDA has agreed to review an application for a biosimilar of Simponi. The companies said they expect a decision from the FDA in the fourth quarter.
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2 months ago | 
pnhnews.com | Marisa Wexler MS |Esteban Cerezo |Brandi Lewis |Patricia Inacio
Long-term treatment with Ultomiris (ravulizumab) reduced the risk of death by five times in people with paroxysmal nocturnal hemoglobinuria (PNH), and also tended to eliminate the need for blood transfusions and to improve patients’ quality of life, a new analysis suggests.
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2 months ago | 
pnhnews.com | Katherine Poinsatte |Andrea Lobo |Esteban Cerezo |Marisa Wexler MS
Voydeya (danicopan) effectively and safely treated paroxysmal nocturnal hemoglobinuria (PNH) in people with significant extravascular hemolysis, when used as an add-on therapy to Ultomiris (ravulizumab) or Soliris (eculizumab). That’s according to long-term data from the Phase 3 ALPHA trial (NCT04469465), which found that dual treatment with Voydeya and a C5 inhibitor could reduce extravascular hemolysis, while keeping intravascular hemolysis under control.
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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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Nov 25, 2024 | 
pnhnews.com | Andrea Lobo |Marisa Wexler MS |Margarida Maia |Steve Bryson
Voydeya (danicopan) — whether given as a single therapy or as an add-on to standard treatment — is safe and effective in treating people with paroxysmal nocturnal hemoglobinuria (PNH). That’s according to a systematic review and meta-analysis of data from four international clinical trials that tested the PNH treatment in more than 75 people with the rare acquired disease, which is marked by the destruction of blood cells.
