Paroxysmal Nocturnal Hemoglobinuria
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CLINICAL_TRIALS trial_result · 2025-03-12Clinical Trial Update: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study [Phase 3]
Phase 3 long-term safety and efficacy surveillance trial of Coversin (rVA576) in paroxysmal nocturnal hemoglobinuria was terminated with res…
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CLINICAL_TRIALS other · 2025-03-11Clinical Trial Update: Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy [Phase 2]
A Phase 2 clinical trial update for NM8074 in adults with paroxysmal nocturnal hemoglobinuria who are naive to complement inhibitors. The st…
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CLINICAL_TRIALS trial_result · 2025-03-07Clinical Trial Update: BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor Therapy [Phase 2]
Phase 2 study of BCX9930 in Paroxysmal nocturnal hemoglobinuria patients with inadequate response to C5 inhibitors has been terminated, with…
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CLINICAL_TRIALS trial_result · 2025-03-03Clinical Trial Update: Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies [NA]
A hematopoietic stem cell transplant trial for high-risk hemoglobinopathies has been terminated; the conditions studied include sickle cell …
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CLINICAL_TRIALS trial_result · 2025-02-28Clinical Trial Update: Study on Mass Balance of [14C]HSK39297 in Healthy Chinese Adult Male Subjects [Phase 1]
Phase 1 mass-balance study of radiolabeled HSK39297 in healthy Chinese adult male volunteers has been completed, with Paroxysmal nocturnal h…
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CLINICAL_TRIALS research · 2025-02-27Clinical Trial Update: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of of VSA012 in Subjects with Paroxysmal Nocturnal Hemoglobinuria(PNH)
This is an early-phase clinical trial update evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary effica…
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CLINICAL_TRIALS research · 2025-02-20Clinical Trial Update: Global PNH Patient Registry
Global patient registry for Paroxysmal Nocturnal Hemoglobinuria is currently recruiting. The registry aims to collect natural history and ou…
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FDA approval · 2025-01-30FDA Approved: BKEMV — Supplemental Application (Amgen Inc)
FDA approved BKEMV (eculizumab-aeeb) under Amgen’s supplemental application, making a biosimilar to eculizumab available. The product is int…
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CLINICAL_TRIALS trial_result · 2025-01-28Clinical Trial Update: A Retrospective Non-Interventional Study to Evaluate the Impact of Eculizumab in Korean PNH Patients
A retrospective non-interventional study evaluates the impact of eculizumab in Korean patients with paroxysmal nocturnal hemoglobinemia (PNH…
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CLINICAL_TRIALS trial_result · 2025-01-27Clinical Trial Update: Observational of PNH Type Cells in Korean Patients With Bone Marrow Failure Syndrome and Having Hemolytic PNH
This completed observational clinical trial from Handok Inc. investigates PNH type cells in Korean patients with bone marrow failure syndrom…
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CLINICAL_TRIALS trial_result · 2025-01-16Clinical Trial Update: Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy [Phase 2]
A Phase 2 clinical trial evaluated the combination of pozelimab and cemdisiran in adults with paroxysmal nocturnal hemoglobinuria who previo…
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CLINICAL_TRIALS trial_result · 2025-01-08Clinical Trial Update: Dose Escalation of BCX10013 in Participants with Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 1]
This Phase 1 dose-escalation trial of BCX10013 in Paroxysmal nocturnal hemoglobinuria has completed its dose-escalation portion.
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CLINICAL_TRIALS trial_result · 2025-01-03Clinical Trial Update: BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor Therapy [Phase 2]
A Phase 2 trial evaluating BCX9930 for Paroxysmal Nocturnal Hemoglobinuria in participants not receiving other complement inhibitors was ter…
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CLINICAL_TRIALS research · 2024-12-20Clinical Trial Update: Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria [Phase 2]
A Phase 2 clinical trial assessing long-term safety and tolerability of HSK39297 in patients with paroxysmal nocturnal hemoglobinuria is cur…
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CLINICAL_TRIALS trial_result · 2024-12-13Clinical Trial Update: A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
Phase 2 long-term safety study update for BCX9930 in participants with Paroxysmal nocturnal hemoglobinuria. The trial was terminated and res…
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CLINICAL_TRIALS trial_result · 2024-11-01Clinical Trial Update: Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal Hemoglobinuria [Phase 2]
A Phase 2 proof-of-concept study evaluating the efficacy, safety, and pharmacokinetics of LFG316 in patients with Paroxysmal Nocturnal Hemog…
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CLINICAL_TRIALS research · 2024-10-28Clinical Trial Update: To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104 [Phase 2]
A Phase 2 clinical trial is evaluating KP104 for safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy in Paroxysmal noctur…
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FDA approval · 2024-10-23FDA Approved: BKEMV — Supplemental Application (Amgen Inc)
The FDA approved a supplemental application for BKEMV (eculizumab-aeeB) from Amgen, updating its labeling and indications for this eculizuma…
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CLINICAL_TRIALS trial_result · 2024-10-15Clinical Trial Update: Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria [Phase 2]
This update reports the completion of a Phase 2 proof-of-concept trial evaluating the efficacy and safety of HRS-5965 in patients with parox…
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CLINICAL_TRIALS research · 2024-10-08Clinical Trial Update: Paroxysmal Nocturnal Hemoglobinuria in ESUS & ETUS
An update on a clinical trial exploring paroxysmal nocturnal hemoglobinuria in embolic strokes of undetermined source (ESUS/ETUS) and relate…
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FDA approval · 2024-10-07FDA Approved: EMPAVELI — Supplemental Application (Apellis Pharmaceuticals, Inc.)
FDA approved a supplemental application for EMPAVELI (pegcetacoplan) submitted by Apellis Pharmaceuticals, Inc.
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FDA approval · 2024-09-19FDA Approved: SOLIRIS — Supplemental Application (Alexion Pharmaceuticals Inc.)
The FDA approved a supplemental application for SOLIRIS (eculizumab) filed by Alexion Pharmaceuticals, resulting in updated labeling that ma…
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CLINICAL_TRIALS trial_result · 2024-09-19Clinical Trial Update: Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria [Phase 3]
Phase 3 study evaluating the efficacy and safety of the combination of Pozelimab and Cemdisiran versus continued Eculizumab or Ravulizumab i…
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CLINICAL_TRIALS trial_result · 2024-08-12Clinical Trial Update: Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab [Phase 4]
Phase 4 study evaluating Ravulizumab in adults with paroxysmal nocturnal hemoglobinuria who are currently on high-dose eculizumab; results p…
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CLINICAL_TRIALS trial_result · 2024-08-09Clinical Trial Update: Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy [Phase 2]
A Phase 2 study evaluating the oral Factor D inhibitor ALXN2050 as monotherapy in paroxysmal nocturnal hemoglobinuria was terminated, with r…