Paroxysmal Nocturnal Hemoglobinuria
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Sign up freeTop drugs studied for this disease
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FDA approval · 2026-03-26FDA Approved: BKEMV — Supplemental Application (Amgen Inc)
FDA approved a supplemental application for BKEMV (eculizumab-aeeB) from Amgen.
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CLINICAL_TRIALS research · 2026-03-18Clinical Trial Update: A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works [Phase 3]
Phase 3 study evaluating long-term safety and efficacy of Pozelimab plus Cemdisiran in adults with paroxysmal nocturnal hemoglobinuria; recr…
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CLINICAL_TRIALS research · 2026-03-17Clinical Trial Update: A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor [Phase 1, Phase 2]
Phase 1/2 first-in-human trial evaluating ALN-CFB, an siRNA against complement factor B, for adults with paroxysmal nocturnal hemoglobinuria…
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CLINICAL_TRIALS trial_result · 2026-03-09Clinical Trial Update: Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) [Phase 2]
This update reports the completion of a Phase 2 study evaluating a treosulfan-based conditioning regimen before bone marrow or blood stem ce…
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CLINICAL_TRIALS research · 2026-03-06Clinical Trial Update: Danicopan Early Access Program
An early access program for danicopan is described for patients with paroxysmal nocturnal hemoglobinuria (PNH), including those with extrava…
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CLINICAL_TRIALS research · 2026-02-24Clinical Trial Update: A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment [Phase 3]
Phase 3 clinical trial evaluating the safety and efficacy of a pozelimab and cemdisiran combination in adult patients with paroxysmal noctur…
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EMA approval · 2026-02-19EMA Authorised: Soliris (Eculizumab) — Alexion Europe SAS
The EMA authorised Soliris (eculizumab) in the European Union for paroxysmal nocturnal hemoglobinuria. The decision carries orphan medicine …
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EMA approval · 2026-02-18EMA Authorised: Epysqli (Eculizumab) — Samsung Bioepis NL B.V.
EMA authorised Epysqli, a biosimilar of eculizumab, by Samsung Bioepis NL B.V., for paroxysmal nocturnal hemoglobinuria.
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CLINICAL_TRIALS research · 2026-02-18Clinical Trial Update: A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy
This document reports an update on a clinical trial testing iptacopan in Korean patients with Paroxysmal Nocturnal Hemoglobinuria or C3 glom…
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CLINICAL_TRIALS research · 2026-02-17Clinical Trial Update: Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data
Clinical trial update analyzing long-term safety of Danicopan as add-on therapy in paroxysmal nocturnal hemoglobinuria using IPIG-Registry d…
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CLINICAL_TRIALS research · 2026-02-17Clinical Trial Update: Study to Assess Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Healthy Volunteers and Participants With Paroxysmal Nocturnal Hemoglobinuria [Phase 1, Phase 2]
This entry reports on a Phase 1/2 clinical trial evaluating crovalimab in healthy volunteers and patients with paroxysmal nocturnal hemoglob…
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CLINICAL_TRIALS research · 2026-02-17Clinical Trial Update: Long-term Safety of Danicopan: IPIG Registry-based Cohort Study
Clinical trial update on the long-term safety of danicopan in a registry-based cohort for paroxysmal nocturnal hemoglobinuria, currently act…
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CLINICAL_TRIALS research · 2026-02-12Clinical Trial Update: National Longitudinal Cohort of Hematological Diseases
Recruiting for a national longitudinal cohort study of hematological diseases sponsored by the Institute of Hematology & Blood Diseases Hosp…
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EMA approval · 2026-02-10EMA Authorised: Piasky (Crovalimab) — Roche Registration GmbH
The European Medicines Agency authorised Piasky (crovalimab) by Roche Registration GmbH for the treatment of paroxysmal nocturnal hemoglobin…
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CLINICAL_TRIALS research · 2026-02-04Clinical Trial Update: SLN12140 in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China [Phase 2]
Phase 2 clinical trial update for SLN12140 in adults with paroxysmal nocturnal hemoglobinuria in China; the study is not yet recruiting.
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CLINICAL_TRIALS research · 2026-01-28Clinical 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]
Phase 3 trial update evaluating safety, pharmacokinetics, and efficacy of crovalimab versus eculizumab in participants with paroxysmal noctu…
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EMA approval · 2026-01-26EMA Authorised: Aspaveli (pegcetacoplan) — Swedish Orphan Biovitrum AB (publ)
The European Medicines Agency authorised Aspaveli (pegcetacoplan) by Swedish Orphan Biovitrum AB (publ) for the treatment of paroxysmal noct…
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CLINICAL_TRIALS trial_result · 2026-01-21Clinical Trial Update: Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 3]
This Phase 3 trial update assesses the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of ravulizumab in Chinese ad…
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CLINICAL_TRIALS research · 2026-01-20Clinical Trial Update: Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
Completed status for the Paroxysmal nocturnal hemoglobinuria (PNH) Registry, an observational study sponsored by Alexion Pharmaceuticals to …
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CLINICAL_TRIALS research · 2026-01-16Clinical Trial Update: Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in PNH Patients. [Phase 2]
Phase 2 clinical trial update of LP-005 in paroxysmal nocturnal hemoglobinuria, reporting on efficacy, safety, and pharmacokinetics; the stu…
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EMA approval · 2026-01-15EMA Authorised: Bekemv (Eculizumab) — Amgen Technology (Ireland) UC
The EMA authorised Bekemv (eculizumab) from Amgen Technology (Ireland) UC for paroxysmal nocturnal hemoglobinuria.
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EMA approval · 2026-01-13EMA Authorised: Voydeya (danicopan) — Alexion Europe SAS
The European Medicines Agency authorised Voydeya (danicopan) for paroxysmal nocturnal hemoglobinuria, designated as an orphan medicine, with…
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CLINICAL_TRIALS trial_result · 2026-01-08Clinical Trial Update: Efficacy and Safety of HRS-5965 in Patients With PNH Who Are Still Anemia After Anti-C5 Antibody Treatment [Phase 3]
Phase 3 clinical trial evaluating the efficacy and safety of HRS-5965 in Paroxysmal nocturnal hemoglobinuria patients who remain anemic afte…
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CLINICAL_TRIALS trial_result · 2025-12-24Clinical Trial Update: Comparison of HRS-5965 Capsules and Tablets in Healthy Subjects [Phase 1]
Phase 1 trial comparing capsule and tablet formulations of HRS-5965 in healthy subjects has been completed.
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CLINICAL_TRIALS trial_result · 2025-12-24Clinical Trial Update: Long-term Safety and Tolerability of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria [Phase 2]
This clinical trial update reports on the long-term safety and tolerability evaluation of HRS-5965 in patients with Paroxysmal Nocturnal Hem…