Paroxysmal Nocturnal Hemoglobinuria
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CLINICAL_TRIALS trial_result · 2025-08-20Clinical Trial Update: A Clinical Study to Evaluate the Effect of Food on the Pharmacokinetic Profiles of HRS-5965 Capsules in Healthy Subjects [Phase 1]
Phase 1 study evaluating the effect of food on the pharmacokinetics of HRS-5965 capsules in healthy subjects has completed. Conditions of in…
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CLINICAL_TRIALS research · 2025-08-06Clinical Trial Update: Hematological Disorders in EHPVO Patients
A clinical trial update describes a study on hematological disorders in patients with Extrahepatic portal vein obstruction and thrombophilia…
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CLINICAL_TRIALS trial_result · 2025-07-30Clinical Trial Update: A Study of MY008211A in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
A Phase 2 clinical trial of MY008211A in adult patients with paroxysmal nocturnal hemoglobinuria has completed.
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CLINICAL_TRIALS research · 2025-07-29Clinical Trial Update: Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
A Phase 2 clinical trial investigating the safety and efficacy of MY008211A in patients with paroxysmal nocturnal hemoglobinuria (PNH) is li…
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FDA approval · 2025-07-28FDA Approved: EMPAVELI — Supplemental Application (Apellis Pharmaceuticals, Inc.)
FDA approved a supplemental application for EMPAVELI (pegcetacoplan) from Apellis Pharmaceuticals.
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CLINICAL_TRIALS research · 2025-07-16Clinical Trial Update: Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF [NA]
Clinical trial update on unrelated and partially matched related donor PSCT with TCR αβ depletion for bone marrow failure conditions; the st…
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CLINICAL_TRIALS trial_result · 2025-06-29Clinical Trial Update: A Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH） [Phase 2]
Phase 2 clinical trial evaluating the efficacy and safety of HSK39297 in patients with paroxysmal nocturnal hemoglobinuria has completed.
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FDA approval · 2025-06-27FDA Approved: VOYDEYA — Supplemental Application (Alexion Pharmaceuticals Inc.)
FDA approved a supplemental application for VOYDEYA (danicopan) by Alexion Pharmaceuticals to treat paroxysmal nocturnal hemoglobinuria.
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CLINICAL_TRIALS trial_result · 2025-06-25Clinical Trial Update: Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy [Phase 2]
This Phase 2 trial assesses the combination of pozelimab and cemdisiran in adults with paroxysmal nocturnal hemoglobinuria who switched from…
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CLINICAL_TRIALS research · 2025-06-19Clinical Trial Update: MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure. [Phase 2]
Phase 2 trial evaluating hematopoietic cell transplantation with reduced-intensity conditioning and post-transplant cyclophosphamide for sev…
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CLINICAL_TRIALS trial_result · 2025-06-12Clinical Trial Update: Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
A completed clinical trial update reports real-world use of ravulizumab in Italian patients with paroxysmal nocturnal hemoglobinuria.
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CLINICAL_TRIALS research · 2025-06-11Clinical Trial Update: A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
A Phase 2 clinical trial update indicates that pegcetacoplan for pediatric patients with paroxysmal nocturnal hemoglobinuria is currently re…
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FDA approval · 2025-06-10FDA Approved: EMPAVELI — Supplemental Application (Apellis Pharmaceuticals, Inc.)
The FDA approved a supplemental application for EMPAVELI (pegcetacoplan) from Apellis Pharmaceuticals.
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CLINICAL_TRIALS trial_result · 2025-05-29Clinical Trial Update: An Extension Clinical Study of BCD-148 for the Treatment of Patients With PNH Previously Treated in Clinical Study No. BCD-148-2/NOCTURN [Phase 3]
An extension Phase 3 study of BCD-148 in patients with paroxysmal nocturnal hemoglobinuria (PNH) who previously participated in BCD-148-2/NO…
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CLINICAL_TRIALS research · 2025-05-18Clinical Trial Update: A Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
Phase II clinical trial evaluating the efficacy and safety of XH-S003 capsules in patients with paroxysmal nocturnal hemoglobinuria, current…
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CLINICAL_TRIALS trial_result · 2025-05-15Clinical Trial Update: Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the IPIG PNH Registry
Post-authorization safety study evaluating iptacopan in adults with paroxysmal nocturnal hemoglobinuria using data from the IPIG PNH registr…
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FDA approval · 2025-05-14FDA Approved: PIASKY — Supplemental Application (Genentech, Inc.)
FDA approved a supplemental application for PIASKY (crovalimab) from Genentech, Inc., resulting in an updated labeling per the approval.
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CLINICAL_TRIALS research · 2025-05-14Clinical Trial Update: A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This clinical trial update notes that a real-world effectiveness study of pegcetacoplan for Paroxysmal nocturnal hemoglobinuria is currently…
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CLINICAL_TRIALS research · 2025-05-09Clinical Trial Update: Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody Treatment [Phase 3]
Phase 3 clinical trial evaluating the safety and efficacy of MY008211A in patients with Paroxysmal nocturnal hemoglobinuria who have residua…
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CLINICAL_TRIALS trial_result · 2025-05-04Clinical Trial Update: Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms [Phase 2]
Phase 2 results for coversin in paroxysmal nocturnal hemoglobinuria patients who are resistant to eculizumab due to C5 polymorphisms; the tr…
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CLINICAL_TRIALS research · 2025-04-18Clinical Trial Update: Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria [Phase 2, Phase 3]
A long-term safety and tolerability study of MY008211A tablets in paroxysmal nocturnal hemoglobinuria is enrolling in Phase 2 and Phase 3. T…
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CLINICAL_TRIALS trial_result · 2025-04-16Clinical Trial Update: Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China [Phase 3]
This clinical trial update reports on a Phase 3 study evaluating eculizumab in adult participants with paroxysmal nocturnal hemoglobinuria i…
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CLINICAL_TRIALS trial_result · 2025-04-09Clinical Trial Update: Treating Paroxysmal Nocturnal Haemoglobinuria Patients With rVA576 [Phase 3]
This Clinical Trials update notes completion of a Phase 3 study of rVA576 for treating paroxysmal nocturnal hemoglobinuria (PNH).
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CLINICAL_TRIALS research · 2025-03-30Clinical Trial Update: Study of NM8074 in Soliris-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
Phase 2 study evaluating NM8074 in patients with paroxysmal nocturnal hemoglobinuria who are already treated with Soliris; the trial is not …
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FDA approval · 2025-03-12FDA Approved: EPYSQLI — Supplemental Application (Teva Pharmaceuticals USA, Inc.)
FDA approves Teva's supplemental application for EPYSQLI (eculizumab-aagh), a biosimilar to eculizumab.