RAIN
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News
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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APLASTIC ANEMIA | Incidence and Clinical Management in Spain Consistent with Other Studies
The study was carried out through a multicenter, ambispective, observational approach encompassing 7 Spanish hospitals, catering to roughly 3.91 million individuals, over the span from 2010 to 2019. The research…
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MYELODYSPLASTIC SYNDROMES | Advantages of Iron Chelation Revealed in Analysis of Low-Risk MDS
A recent groundbreaking study sheds new light on the effectiveness of iron chelation therapy (ICT) for patients suffering from low-risk myelodysplastic syndromes (LR-MDS). By delving into the impact of ICT…
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COLD AGGLUTININ DISEASE | ANX1502 Passes Phase 1 Trial, Proving Safe for Healthy Volunteers
Administered orally as a liquid suspension, ANX1502 demonstrated good tolerability, with no serious side effects reported. The drug’s pharmacokinetics also supported twice-daily dosing for a future proof-of-concept study. “We are…
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wAIHA | Fostamatinib May Induce Durable Hemoglobin Response in Subset of Patients
Interestingly, this positive outcome was predominantly observed among participants hailing from North America, Australia, or Western Europe, while those from Eastern Europe didn’t show the same results. The current treatment…
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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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CLINICAL NEWS | Agios Meets Clinical Proof-of-Concept in Anaemia Treatment for Adults with Lower-Risk MDS
On Monday, the specialized disease company announced the successful achievement of “proof-of-concept” in a Phase 2a trial involving AG-946, a pyruvate kinase (PK) activator aimed at treating anemia in patients…
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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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