#PNH
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News
PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | Patients May See Improved Outcomes with Ravulizumab
The findings showed that patients treated with ravulizumab demonstrated better adherence and persistence compared to those on other complement inhibitors. The ADVANTAGE study, a retrospective longitudinal analysis, included data from…
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REGULATORY APPROVAL | PiaSky Becomes the First Monthly Treatment for PNH in the EU
Paroxysmal Nocturnal Haemoglobinuria (PNH) is a rare and life-threatening blood condition where red blood cells are destroyed by the complement system – part of the innate immune system – causing…
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NEW APPROVAL | FDA Endorses Piasky for Paroxysmal Nocturnal Hemoglobinuria
Piasky, developed by Roche, is a monoclonal antibody that targets complement protein C5. Crovalimab binds to C5 to prevent its cleavage, thus preventing the formation of the membrane attack complex…
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REGULATORY NEWS | EU Backs Revolutionary Oral Monotherapy for Adult PNH Patients
The decision was hailed as a first step toward enabling patient access in European Union countries following a March 21 meeting of the Committee for Medicinal Products for Human Use…
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REGULATORY NEWS | AstraZeneca’s Voydeya Recommended for PNH Treatment in EU
Voydeya is a first-in-class, oral, Factor D inhibitor developed as an add-on to standard-of care Ultomiris (ravulizumab) or Soliris (eculizumab) to address the needs of the approximately 10-20% of patients with PNH who…
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PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | Ruxoprubart Scores FDA Orphan Drug Designation for PNH Treatment
FDA’s recognition of Ruxoprubart as an orphan drug for PNH underscores its potential to fulfill a crucial need for individuals grappling with this disease condition. Ruxoprubart is currently undergoing a…
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NEWS | ICER Releases Draft Evidence Report on Treatment for Paroxysmal Nocturnal Hemoglobinuria
This preliminary draft marks the midpoint of ICER’s eight-month process of assessing these treatments, and the findings within this document should not be interpreted to be ICER’s final conclusions. Register…
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PHARMA NEWS | FDA Approves Iptacopan for Paroxysmal Nocturnal Hemoglobinuria
Fabhalta is a Factor B inhibitor that acts proximally in the alternative complement pathway of the immune system, providing comprehensive control of red blood cell (RBC) destruction within and outside…
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PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | LDH Improvements Lead to Better Outcomes in Fatigue and QoL
PNH is a rare anaemia known for causing blood cell destruction, which can be shown by LDH levels. The study compared two drugs, eculizumab and ravulizumab, to see how they…
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EHA2023 | Crovalimab Appears Non-inferior to Eculizumab in PNH, Data Shows
At the 2023 EHA Congress, researchers presented data from the ongoing COMMODORE 2 study, which was designed to determine whether crovalimab is non-inferior to eculizumab for multiple clinical endpoints among…
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