Paroxysmal Nocturnal Hemoglobinuria

• CLINICAL_TRIALS trial_result · 2025-12-23
  Clinical Trial Update: Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis [Phase 3]

  Phase 3 clinical trial update indicates danicopan as an add-on to ravulizumab or eculizumab in pediatric patients with paroxysmal nocturnal …

• CLINICAL_TRIALS trial_result · 2025-12-22
  Clinical Trial Update: A Phase III Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor Therapy [Phase 3]

  Phase III study evaluating HSK39297 in paroxysmal nocturnal hemoglobinuria patients naive to complement inhibitor therapy has completed. The…

• CLINICAL_TRIALS research · 2025-12-22
  Clinical Trial Update: Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study

  A clinical trial update describing a secondary research study that analyzes molecular and clinical aspects of bone marrow failure and VEXAS …

• EMA approval · 2025-12-19
  EMA Authorised: Fabhalta (iptacopan hydrochloride monohydrate) — Novartis Europharm Limited

  The European Medicines Agency authorized Fabhalta (iptacopan hydrochloride monohydrate) from Novartis Europharm Limited for paroxysmal noctu…

• CLINICAL_TRIALS trial_result · 2025-12-18
  Clinical Trial Update: A Real-world, Multi-center, Prospective, Observational Study for PNH in China

  A real-world, multi-center, prospective observational study on paroxysmal nocturnal hemoglobinuria (PNH) conducted in China has completed, s…

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• CLINICAL_TRIALS trial_result · 2025-12-15
  Clinical Trial Update: Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan. [Phase 3]

  Phase 3 single-arm, open-label trial evaluating switching from anti-C5 therapy to iptacopan for 24 weeks in patients with paroxysmal nocturn…

• CLINICAL_TRIALS trial_result · 2025-12-15
  Clinical Trial Update: A Study of Single and Multiple Doses of LP-005 in Healthy Adult Participants [Phase 1]

  Longbio Pharma has completed a Phase 1 trial evaluating single and multiple doses of LP-005 in healthy adult participants to explore its saf…

• CLINICAL_TRIALS research · 2025-12-05
  Clinical Trial Update: Managed Access Programs for LNP023, Iptacopan

  This clinical trial update notes the availability of managed access programs for iptacopan (LNP023) for patients with C3 glomerulopathy and …

• CLINICAL_TRIALS research · 2025-12-05
  Clinical Trial Update: Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]

  A Phase 2 clinical trial evaluating the efficacy, safety, and pharmacokinetics of LP-005 injection in individuals with paroxysmal nocturnal …

• CLINICAL_TRIALS research · 2025-12-01
  Clinical Trial Update: A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China

  A multi-center ambispective cohort study in China evaluating the impact of iptacopan for adults with paroxysmal nocturnal hemoglobinuria. Th…

• CLINICAL_TRIALS research · 2025-12-01
  Clinical Trial Update: A Phase Ib, Multicenter, Open-Label Study of Multiple-Dose EA5 in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 1]

  A Phase Ib, multicenter, open-label study to evaluate multiple-dose EA5 in adults with paroxysmal nocturnal hemoglobinuria is currently recr…

• FDA approval · 2025-11-24
  FDA Approved: BKEMV — Supplemental Application (Amgen Inc)

  The FDA approved a supplemental application for BKEMV (eculizumab-aeeB) from Amgen, a biosimilar to eculizumab.

• CLINICAL_TRIALS trial_result · 2025-11-10
  Clinical Trial Update: A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors [Phase 3]

  This Phase 3 trial compares crovalimab against eculizumab in adults with paroxysmal nocturnal hemoglobinuria who have not previously receive…

• CLINICAL_TRIALS trial_result · 2025-11-10
  Clinical Trial Update: A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors [Phase 3]

  Phase 3 trial comparing crovalimab to eculizumab in treatment-naïve PNH patients; study status active/not recruiting with results posted in …

• CLINICAL_TRIALS trial_result · 2025-10-24
  Clinical Trial Update: Long-term Safety of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria [Phase 2]

  Phase 2 long-term safety update for BCX9930 in adults with paroxysmal nocturnal hemoglobinuria; the study is terminated and results have bee…

• EMA approval · 2025-10-22
  EMA Authorised: Ultomiris (ravulizumab) — Alexion Europe SAS

  The European Medicines Agency authorised Ultomiris (ravulizumab) for paroxysmal nocturnal hemoglobinuria, under the responsibility of Alexio…

• CLINICAL_TRIALS withdrawal · 2025-10-20
  Clinical Trial Update: Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria

  The clinical trial update indicates that compassionate-use access to the pozelimab and cemdisiran combination therapy for Paroxysmal nocturn…

• CLINICAL_TRIALS trial_result · 2025-10-01
  Clinical Trial Update: Safety and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria Patients With a Sub-optimal Response to Ravulizumab [Phase 2]

  Phase 2 trial evaluating OMS906 in paroxysmal nocturnal hemoglobinuria patients with suboptimal response to ravulizumab reports safety and e…

• CLINICAL_TRIALS research · 2025-09-23
  Clinical Trial Update: A Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients [Phase 2]

  This is a Phase 2 study evaluating NTQ5082 capsules for paroxysmal nocturnal hemoglobinuria. The study is currently active but not recruitin…

• CLINICAL_TRIALS research · 2025-09-17
  Clinical Trial Update: A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients [Phase 3]

  A Phase III study of NTQ5082 capsules for paroxysmal nocturnal hemoglobinuria is planned, with the trial currently not yet recruiting.

• CLINICAL_TRIALS research · 2025-09-17
  Clinical Trial Update: A Long-term Efficacy and Safety of NTQ5082 Capsules [Phase 3]

  This update describes a Phase 3 long-term efficacy and safety study of NTQ5082 capsules for paroxysmal nocturnal hemoglobinuria, currently n…

• CLINICAL_TRIALS trial_result · 2025-09-11
  Clinical Trial Update: A Study to Evaluate the Efficacy and Safety of Oral HRS-5965 in Adult Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy [Phase 3]

  A Phase 3 clinical trial update evaluating the efficacy and safety of oral HRS-5965 in adults with paroxysmal nocturnal hemoglobinuria who h…

• CLINICAL_TRIALS research · 2025-09-03
  Clinical Trial Update: Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic Impairment [Phase 1]

  Phase 1 study evaluating the pharmacokinetics, safety, and pharmacodynamics of the investigational drug HSK39297 in subjects with hepatic im…

• FDA approval · 2025-09-02
  FDA Approved: EPYSQLI — Supplemental Application (Teva Pharmaceuticals USA, Inc.)

  FDA approves a supplemental application for EPYSQLI (eculizumab-aagh) from Teva Pharmaceuticals USA, Inc.

• CLINICAL_TRIALS research · 2025-08-27
  Clinical Trial Update: Home Reported Outcomes in PNH

  Clinical trial update indicating a Novartis-sponsored study on home-reported outcomes in paroxysmal nocturnal hemoglobinuria is currently re…