Paroxysmal Nocturnal Hemoglobinuria
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CLINICAL_TRIALS trial_result · 2022-05-09Clinical Trial Update: A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria [Phase 3]
Phase 3 study of Ravulizumab in children and adolescents with paroxysmal nocturnal hemoglobinuria has completed and results were posted.
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CLINICAL_TRIALS trial_result · 2022-03-25Clinical Trial Update: Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 3]
Phase 3 trial evaluating APL-2 for paroxysmal nocturnal hemoglobinuria has completed with results posted.
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CLINICAL_TRIALS trial_result · 2022-02-17Clinical Trial Update: First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNH [Phase 1, Phase 2]
First-in-human study of BCX9930 in healthy volunteers and PNH patients completed, reporting Phase 1/2 results.
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CLINICAL_TRIALS trial_result · 2021-09-01Clinical Trial Update: Efficacy and Safety of Elizaria® vs. Soliris® in Patients With PNH [Phase 3]
Phase 3 trial comparing Elizaria® with Soliris® in patients with Paroxysmal Nocturnal Hemoglobinuria, assessing efficacy and safety results.
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CLINICAL_TRIALS trial_result · 2021-06-25Clinical Trial Update: Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab [Phase 2]
Phase 2 study evaluating danicopan in PNH patients with inadequate response to eculizumab has completed with posted results.
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FDA approval · 2021-06-07FDA Approved: ULTOMIRIS — Supplemental Application (Alexion Pharmaceuticals Inc.)
FDA approved a supplemental application for ULTOMIRIS (ravulizumab) from Alexion Pharmaceuticals, reflecting an update to labeling or expans…
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CLINICAL_TRIALS trial_result · 2021-06-02Clinical Trial Update: Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
Phase 2 study of danicopan in Paroxysmal Nocturnal Hemoglobinuria completed with results posted in 2021.
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FDA approval · 2021-05-14FDA Approved: EMPAVELI — Original Application (Apellis Pharmaceuticals, Inc.)
The FDA approved EMPAVELI (pegcetacoplan) from Apellis Pharmaceuticals as a treatment for adults with paroxysmal nocturnal hemoglobinuria wh…
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CLINICAL_TRIALS trial_result · 2021-02-12Clinical Trial Update: Clinical Trial of BCD-148 and Soliris® for the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria [Phase 3]
Phase 3 trial update for BCD-148 and Soliris in paroxysmal nocturnal hemoglobinuria (PNH) has completed.
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CLINICAL_TRIALS trial_result · 2021-01-11Clinical Trial Update: Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects. [Phase 1]
Pilot Phase 1 trial evaluating safety, preliminary efficacy, and pharmacokinetics of subcutaneous pegylated complement inhibitor pegcetacopl…
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CLINICAL_TRIALS trial_result · 2021-01-08Clinical Trial Update: A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH [Phase 1]
Phase I study evaluating the safety of pegcetacoplan (APL-2) as an add-on to standard of care in paroxysmal nocturnal hemoglobinuria (PNH).
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CLINICAL_TRIALS trial_result · 2020-12-28Clinical Trial Update: A Safety and Immunogenicity Study in Long-term Treatment of Eculizumab (JSC "GENERIUM", Russian Federation) [Phase 3]
A Phase 3 safety and immunogenicity trial of long-term eculizumab in patients with Paroxysmal Nocturnal Hemoglobinuria has completed.
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CLINICAL_TRIALS trial_result · 2020-12-22Clinical Trial Update: A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNH [Phase 2]
Phase IIa trial evaluating subcutaneous pegcetacoplan (APL-2) for safety, efficacy, and pharmacokinetics in patients with paroxysmal nocturn…
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FDA approval · 2020-10-09FDA Approved: ULTOMIRIS — Supplemental Application (Alexion Pharmaceuticals Inc.)
The FDA approved a supplemental application for Ultomiris (ravulizumab) submitted by Alexion Pharmaceuticals, resulting in changes to the la…
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CLINICAL_TRIALS trial_result · 2020-03-30Clinical Trial Update: A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH [Phase 1, Phase 2]
Phase 1/2 trial investigating ALN-CC5 in healthy volunteers and patients with Paroxysmal Nocturnal Hemoglobinuria (PNH); study completed wit…
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CLINICAL_TRIALS trial_result · 2020-03-10Clinical Trial Update: Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients [Phase 2]
Phase 2 trial of Zilucoplan (RA101495) for Paroxysmal Nocturnal Hemoglobinuria has completed with results posted in 2020.
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CLINICAL_TRIALS trial_result · 2020-02-26Clinical Trial Update: Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab [Phase 2]
Phase 2 trial assessing safety and efficacy of Zilucoplan (RA101495) for PNH patients inadequately responding to eculizumab; study completed…
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CLINICAL_TRIALS trial_result · 2019-07-12Clinical Trial Update: E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients [Phase 2]
Phase 2 extension study results for safety and efficacy of eculizumab in patients with paroxysmal nocturnal hemoglobinuria have been complet…
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FDA approval · 2019-06-27FDA Approved: SOLIRIS — Supplemental Application (Alexion Pharmaceuticals Inc.)
The FDA approved a supplemental application for SOLIRIS (eculizumab) filed by Alexion Pharmaceuticals Inc., updating labeling to reflect the…
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CLINICAL_TRIALS trial_result · 2019-03-21Clinical Trial Update: ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab [Phase 3]
Phase 3 trial comparing ravulizumab (ALXN1210) with eculizumab in adults with paroxysmal nocturnal hemoglobinuria who are already receiving …
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CLINICAL_TRIALS trial_result · 2019-03-08Clinical Trial Update: Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory PNH [Phase 4]
Phase 4 clinical trial evaluating sirolimus with low-dose warfarin for refractory paroxysmal nocturnal hemoglobinuria.
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CLINICAL_TRIALS trial_result · 2019-02-18Clinical Trial Update: Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
Phase 2 open-label, multiple ascending dose study of ravulizumab (ALXN1210) in participants with paroxysmal nocturnal hemoglobinuria (PNH) h…
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CLINICAL_TRIALS trial_result · 2019-02-12Clinical Trial Update: ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 3]
A Phase 3 trial compares ravulizumab (ALXN1210) with eculizumab in treatment-naïve adults with paroxysmal nocturnal hemoglobinuria; results …
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CLINICAL_TRIALS trial_result · 2019-01-18Clinical Trial Update: Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria [Phase 2]
A Phase 2 study evaluated high-dose cyclophosphamide in patients with severe aplastic anemia and paroxysmal nocturnal hemoglobinuria. The tr…
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FDA approval · 2018-12-21FDA Approved: ULTOMIRIS — Original Application (Alexion Pharmaceuticals Inc.)
FDA approved Ultomiris (ravulizumab) through the original application for treatment of certain complement-mediated diseases, including parox…