Paroxysmal Nocturnal Hemoglobinuria
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Sign up freeTop drugs studied for this disease
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EMA approval · 2026-08-07EMA Authorised: Epysqli (Eculizumab) — Samsung Bioepis NL B.V.
Epilishi (Eculizumab) from Samsung Bioepis NL B.V. has been authorised by EMA for its indicated use in paroxysmal nocturnal hemoglobinuria.
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EMA approval · 2026-07-31EMA Authorised: Aspaveli (pegcetacoplan) — Swedish Orphan Biovitrum AB (publ)
Aspaveli (pegcetacoplan) by Swedish Orphan Biovitrum AB has been authorised by the EMA for paroxysmal nocturnal hemoglobinuria (PNH).
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EMA approval · 2026-07-21EMA Authorised: Ultomiris (ravulizumab) — Alexion Europe SAS
EMA authorises Ultomiris (ravulizumab) for paroxysmal hemoglobinuria (PNH), with Alexion Europe SAS as the marketing authorisation holder.
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CLINICAL_TRIALS trial_result · 2026-06-18Clinical Trial Update: A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH [Phase 3]
Phase 3 long-term safety and efficacy study of danicopan added to a C5 inhibitor in paroxysmal nocturnal hemoglobinuria (PNH) is ongoing; st…
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EMA approval · 2026-06-09EMA Authorised: Ultomiris (ravulizumab) — Alexion Europe SAS
The European Medicines Agency authorised Ultomiris (ravulizumab) for its indicated use under the owner Alexion Europe SAS, with decision dat…
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CLINICAL_TRIALS trial_result · 2026-06-05Clinical Trial Results: Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 3]
Phase 3 trial assessing efficacy, safety, PK/PD, and immunogenicity of Ravulizumab in Chinese adults with Paroxysmal Nocturnal Hemoglobinuri…
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CLINICAL_TRIALS trial_result · 2026-06-05Clinical Trial Update: Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 3]
Phase 3 clinical trial update assessing ravulizumab in Chinese adults with paroxysmal nocturnal hemoglobinuria (PNH), reporting efficacy, sa…
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CLINICAL_TRIALS research · 2026-06-05Clinical Trial Update: Pegcetacoplan Long Term Safety and Efficacy Extension Study [Phase 3]
Phase 3 long-term safety and efficacy extension study for pegcetacoplan in paroxysmal nocturnal hemoglobinuria (PNH) completed.
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CLINICAL_TRIALS trial_result · 2026-06-03Clinical Trial Update: Pegcetacoplan Long Term Safety and Efficacy Extension Study [Phase 3]
Phase 3 long-term safety and efficacy extension study for pegcetacoplan in PNH is active/not recruiting, continuing evaluation of extended o…
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CLINICAL_TRIALS trial_result · 2026-04-28Clinical Trial Update: A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors [Phase 3]
A Phase 3 trial comparing crovalimab to eculizumab in patients with paroxysmal nocturnal hemoglobinuria who are already treated with complem…
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently [Phase 3]
This is a Phase 3 clinical trial evaluating a combination of pozelimab and cemdisiran in adults with paroxysmal nocturnal hemoglobinuria who…
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis [Phase 2]
Phase 2 clinical trial evaluating haplo-identical transplantation with post-transplant cyclophosphamide for GVHD prophylaxis in severe aplas…
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CLINICAL_TRIALS research · 2026-04-22Clinical Trial Update: Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients [Phase 1, Phase 2]
This document reports an update on the safety, tolerability, pharmacokinetics and pharmacodynamics of ADX-038 in healthy participants and Pa…
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CLINICAL_TRIALS trial_result · 2026-04-17Clinical Trial Update: Danicopan PMS in Korea
AstraZeneca’s Danicopan is enrolling in a post-marketing surveillance study in Korea for Paroxysmal Nocturnal Hemoglobinuria.
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CLINICAL_TRIALS research · 2026-04-16Clinical Trial Update: REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
A clinical trial update on the REAL-CARE study evaluating the real-world effectiveness of iptacopan in Italian patients with paroxysmal noct…
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CLINICAL_TRIALS research · 2026-04-16Clinical Trial Update: Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients [Phase 3]
Phase 3 study to assess the pharmacokinetics, safety, and tolerability of iptacopan in pediatric patients with paroxysmal nocturnal hemoglob…
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CLINICAL_TRIALS trial_result · 2026-04-16Clinical Trial Update: A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH [Phase 3]
Phase 3 long-term safety and efficacy study evaluating danicopan as add-on therapy to C5 inhibitors for Paroxysmal nocturnal hemoglobinuria;…
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CLINICAL_TRIALS research · 2026-04-16Clinical Trial Update: Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
This clinical trial update indicates that a study evaluating the safety of Ravulizumab (Ultomiris) in pregnant and postpartum individuals is…
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CLINICAL_TRIALS trial_result · 2026-04-16Clinical Trial Update: Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria [Phase 3]
An ongoing Phase 3 trial is evaluating the long-term safety and tolerability of iptacopan in patients with paroxysmal nocturnal hemoglobinur…
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CLINICAL_TRIALS research · 2026-04-13Clinical Trial Update: Specified Drug-use Surveillance of Fabhalta Capsules
Novartis is conducting a specified drug-use surveillance trial of Fabhalta Capsules in people with Paroxysmal nocturnal hemoglobinuria, curr…
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CLINICAL_TRIALS research · 2026-04-13Clinical Trial Update: Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal Hemoglobinuria [Phase 1, Phase 2]
A clinical trial update for HS-10542 in paroxysmal nocturnal hemoglobinuria, currently in Phase 1/2 and recruiting for safety and efficacy a…
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CLINICAL_TRIALS trial_result · 2026-04-13Clinical Trial Update: Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris [Phase 2]
A Phase 2 study of NM8074 in adults with paroxysmal nocturnal hemoglobinuria (inadequate response to Soliris) is not yet recruiting.
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CLINICAL_TRIALS trial_result · 2026-04-13Clinical Trial Update: NIS PDC the Efficacy and Safety of Iptacopan in Adults in Routine Clinical Practice
This trial update reports the terminated evaluation of iptacopan for efficacy and safety in adults with paroxysmal nocturnal hemoglobinuria …
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CLINICAL_TRIALS research · 2026-04-08Clinical Trial Update: A Phase I Clinical Trial to Evaluate CMS-D017 Following Single and Multiple Doses in Healthy Participants [Phase 1]
This is a Phase 1 clinical trial update evaluating CMS-D017 after single and multiple doses in healthy participants, and the study is curren…
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CLINICAL_TRIALS research · 2026-04-06Clinical Trial Update: Pegcetacoplan Long Term Safety and Efficacy Extension Study [Phase 3]
Long-term safety and efficacy extension study of pegcetacoplan in paroxysmal nocturnal hemoglobinuria (PNH) is ongoing in Phase 3 and is cur…