Paroxysmal Nocturnal Hemoglobinuria
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CLINICAL_TRIALS trial_result · 2024-08-09Clinical Trial Update: To Evaluate the Effect of Single Oral Dose of MY008211A Tablets on QTc Interval in Healthy Subjects [Phase 1]
Phase 1 trial to assess the effect of a single oral dose of MY008211A on QTc interval in healthy subjects; the study is not yet recruiting a…
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CLINICAL_TRIALS research · 2024-07-29Clinical Trial Update: The International PNH Interest Group PNH Registry
This clinical trial update describes an ongoing PNH patient registry led by the International PNH Interest Group to collect data on Paroxysm…
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FDA approval · 2024-07-19FDA Approved: EPYSQLI — Original Application (Teva Pharmaceuticals USA, Inc.)
FDA approved EPYSQLI (eculizumab-aagh) from Teva as a biosimilar to Soliris, for the same indications as the reference product.
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FDA approval · 2024-06-20FDA Approved: PIASKY — Original Application (Genentech, Inc.)
The FDA approved PIASKY (crovalimab) from Genentech via an original BLA, marking its first regulatory approval as a new monoclonal antibody …
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FDA approval · 2024-05-28FDA Approved: BKEMV — Original Application (Amgen Inc)
FDA approved BKEMV (eculizumab-aeeB), a biosimilar to eculizumab, developed by Amgen. The approval covers the same indications as the refere…
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FDA approval · 2024-03-29FDA Approved: VOYDEYA — Original Application (Alexion Pharmaceuticals Inc.)
FDA approved VOYDEYA (danicopan) from Alexion Pharmaceuticals as an oral Factor D inhibitor for adults with paroxysmal nocturnal hemoglobinu…
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CLINICAL_TRIALS trial_result · 2024-03-26Clinical Trial Update: A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal Haemoglobinuria [Phase 3]
Phase 3 trial updating results for SB12 (eculizumab biosimilar) compared to Soliris in paroxysmal nocturnal hemoglobinuria (PNH) has complet…
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FDA approval · 2024-02-08FDA Approved: EMPAVELI — Supplemental Application (Apellis Pharmaceuticals, Inc.)
The FDA approved a supplemental application for EMPAVELI (pegcetacoplan) from Apellis Pharmaceuticals, Inc. to update the product labeling.
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CLINICAL_TRIALS trial_result · 2024-01-03Clinical Trial Update: Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
Phase 2 trial evaluating LNP023 (iptacopan) for safety, efficacy, tolerability, pharmacokinetics, and pharmacodynamics in patients with paro…
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CLINICAL_TRIALS trial_result · 2023-12-18Clinical Trial Update: Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria [Phase 2]
Phase 2 trial evaluating multiple doses of iptacopan (LNP023) in adults with paroxysmal nocturnal hemoglobinuria to assess efficacy, safety,…
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CLINICAL_TRIALS trial_result · 2023-11-28Clinical Trial Update: Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment [Phase 3]
A Phase 3 study evaluating the efficacy and safety of twice-daily oral iptacopan (LNP023) in adult Paroxysmal Nocturnal Hemoglobinuria patie…
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CLINICAL_TRIALS trial_result · 2023-11-15Clinical Trial Update: Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy [Phase 3]
Phase 3 study evaluating the efficacy and safety of twice-daily oral iptacopan (LNP023) in adults with paroxysmal nocturnal hemoglobinuria w…
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FDA approval · 2023-09-28FDA Approved: EMPAVELI — Supplemental Application (Apellis Pharmaceuticals, Inc.)
The FDA approved a supplemental application for EMPAVELI (pegcetacoplan) submitted by Apellis Pharmaceuticals. This action updates the produ…
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CLINICAL_TRIALS trial_result · 2023-07-24Clinical Trial Update: Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA) [Phase 3]
This Phase 3 study evaluates danicopan as an add-on therapy to a C5 inhibitor in Paroxysmal Nocturnal Hemoglobinuria patients with extravasc…
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CLINICAL_TRIALS trial_result · 2023-07-12Clinical Trial Update: Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) [Phase 2]
Phase 2 trial update for Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) has completed with results posted in 2023.
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CLINICAL_TRIALS trial_result · 2023-06-26Clinical Trial Update: Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
Phase 2 trial evaluating REGN3918 for safety and efficacy in paroxysmal nocturnal hemoglobinuria has completed with results posted in 2023.
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CLINICAL_TRIALS trial_result · 2023-06-12Clinical Trial Update: REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability. [Phase 3]
This Phase 3 trial update for REGN3918 in paroxysmal nocturnal hemoglobinuria (PNH) was terminated, with results posted in 2023.
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CLINICAL_TRIALS trial_result · 2023-06-05Clinical Trial Update: A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition [Phase 3]
Phase 3 trial of crovalimab in paroxysmal nocturnal hemoglobinuria patients not previously treated with complement inhibition; results of th…
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CLINICAL_TRIALS trial_result · 2023-05-23Clinical Trial Update: A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH [Phase 3]
Phase 3 trial evaluating ABP 959 versus eculizumab in adults with paroxysmal nocturnal hemoglobinuria has completed and results posted.
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CLINICAL_TRIALS trial_result · 2023-03-14Clinical Trial Update: A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
Phase 2 long-term study of ACH-0144471 in paroxysmal nocturnal hemoglobinuria completed with results posted in 2023.
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FDA approval · 2023-02-08FDA Approved: EMPAVELI — Supplemental Application (Apellis Pharmaceuticals, Inc.)
The FDA approved a supplemental application for EMPAVELI (pegcetacoplan) submitted by Apellis Pharmaceuticals, Inc., resulting in updated la…
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CLINICAL_TRIALS trial_result · 2022-10-27Clinical Trial Update: Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study [Phase 2]
Extension study update for RA101495 in PNH patients who completed a prior Zilucoplan/RA101495 trial; the Phase 2 study is terminated with re…
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CLINICAL_TRIALS trial_result · 2022-10-21Clinical Trial Update: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH [Phase 3]
Phase 3 trial evaluating pegcetacoplan for paroxysmal nocturnal hemoglobinuria has completed with results posted.
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CLINICAL_TRIALS trial_result · 2022-10-20Clinical Trial Update: Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab [Phase 3]
Phase 3 trial comparing subcutaneous versus intravenous ravulizumab in adults with paroxysmal nocturnal hemoglobinuria who are currently tre…
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CLINICAL_TRIALS trial_result · 2022-09-14Clinical Trial Update: A Study on the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of CAN106 in Subjects With PNH [Phase 1, Phase 2]
Clinical trial update on CAN106 assessing safety, tolerability, efficacy, PK, and PD in subjects with paroxysmal nocturnal hemoglobinuria (P…