Hemophilia
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2024-03-27Clinical Trial Update: A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B [Phase 3]
Results from a Phase 3 study of PF-06838435 gene therapy for Hemophilia B have been posted, evaluating efficacy and safety in adult males wi…
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CLINICAL_TRIALS research · 2024-03-27Clinical Trial Update: Intensive Replacement Treatment in Haemophilia Patients With Synovitis [Phase 4]
Phase 4 clinical trial update evaluating intensive replacement treatment in patients with Hemophilia A and synovitis.
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FDA approval · 2024-03-21FDA Approved: DESMOPRESSIN ACETATE — Original Application (Aurobindo Pharma Limited)
FDA approved desmopressin acetate from Aurobindo Pharma Limited under an original abbreviated new drug application (ANDA213095).
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CLINICAL_TRIALS trial_result · 2024-03-20Clinical Trial Update: Pharmacokinetic-guided Dosing of Emicizumab [Phase 4]
Phase 4 clinical trial updating pharmacokinetic-guided dosing of emicizumab in people with Hemophilia A (with and without inhibitors) across…
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CLINICAL_TRIALS trial_result · 2024-03-07Clinical Trial Update: A Study of ADVATE in People With Hemophilia A in India [Phase 4]
A Phase 4 trial of ADVATE in people with Hemophilia A in India has completed with results posted.
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CLINICAL_TRIALS trial_result · 2024-03-07Clinical Trial Update: A Clinical Study of Recombinant Human Coagulation Factor VIII for Injection in Patients With Severe Hemophilia A [Phase 3]
Phase 3 clinical study evaluating recombinant human coagulation factor VIII for injection in severe hemophilia A.
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CLINICAL_TRIALS trial_result · 2024-03-06Clinical Trial Update: Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Participants [Phase 3]
Phase 3 clinical trial update evaluating prophylactic emicizumab versus no prophylaxis in Hemophilia A, with results and pharmacokinetic dat…
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CLINICAL_TRIALS trial_result · 2024-03-05Clinical Trial Update: Comparison of Outcomes Between Low Dose Emicizumab and Factor VIII in Clinically Severe Hemophilia A [Phase 2, Phase 3]
A clinical trial update comparing low-dose emicizumab to Factor VIII in patients with clinically severe hemophilia A without inhibitor, focu…
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CLINICAL_TRIALS trial_result · 2024-02-29Clinical Trial Update: Combination Regimen With Sodium Valproate for Severe Hemophilia: a Single-arm, Phase 1, Pilot Trial. [Phase 1, Phase 2]
A single-arm Phase 1/2 pilot trial evaluating a combination regimen with sodium valproate for severe hemophilia.
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CLINICAL_TRIALS trial_result · 2024-02-28Clinical Trial Update: Phase 3 Clinical Project of Pegylated Recombinant Human Coagulation Factor VIII-Fc Fusion Protein [Phase 3]
Phase 3 clinical trial update for a pegylated recombinant Factor VIII-Fc fusion protein in severe Hemophilia A is currently recruiting.
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CLINICAL_TRIALS trial_result · 2024-02-23Clinical Trial Update: Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors [Phase 1, Phase 2]
A dose-finding study of SPK-8016 gene therapy in Hemophilia A has completed Phase 1/2 testing to support evaluation in patients with FVIII i…
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CLINICAL_TRIALS trial_result · 2024-02-13Clinical Trial Update: Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A [Phase 3]
A Phase 3 study of BIVV001 in pediatric patients with Hemophilia A has completed and posted results on safety, efficacy, and pharmacokinetic…
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FDA label_change · 2024-01-31FDA Approved: HEMLIBRA — Supplemental Application (Genentech, Inc.)
The FDA approved a supplemental application for Hemlibra (emicizumab) from Genentech, updating the product labeling.
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CLINICAL_TRIALS withdrawal · 2024-01-05Clinical Trial Update: Emicizumab PUPs and Nuwiq ITI Study [Phase 3]
A Phase 3 clinical trial comparing emicizumab PUPs and Nuwiq ITI has been withdrawn.
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CLINICAL_TRIALS trial_result · 2024-01-02Clinical Trial Update: Safety and Efficacy of STSP-0601 in Adult Patients With Hemophilia A or B Without Inhibitor [Phase 2]
Phase 2 trial update reporting safety and efficacy of STSP-0601 in adults with hemophilia A or B without inhibitors.
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CLINICAL_TRIALS trial_result · 2023-12-13Clinical Trial Update: An Open Label Study to Determine the Safety and Efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Adolescent and Adult Patients With Hemophilia A. [Phase 3]
A Phase 3 open-label study evaluated the safety and efficacy of recombinant human Coagulation Factor VIII-Fc fusion protein for injection in…
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CLINICAL_TRIALS trial_result · 2023-12-12Clinical Trial Update: Health Related Quality of Life of Youth and Young Adults With Haemophilia A [Phase 4]
A Phase 4 clinical trial evaluated health-related quality of life in youth and young adults with Hemophilia A, completed recruitment.
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CLINICAL_TRIALS trial_result · 2023-11-28Clinical Trial Update: Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) [Phase 3]
Phase 3, single-arm trial evaluating valoctocogene roxaparvovec for Hemophilia A has completed, with results posted on 2023-11-28.
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CLINICAL_TRIALS trial_result · 2023-11-18Clinical Trial Update: rFVIII-Fc (Produced by AryoGen Pharmed Co.) Pharmacokinetic Study [Phase 3]
Phase 3 pharmacokinetic study of rFVIII-Fc produced by AryoGen Pharmed Co. for severe hemophilia A has completed.
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CLINICAL_TRIALS trial_result · 2023-10-05Clinical Trial Update: Preventing Inhibitor Recurrence Indefinitely [Phase 4]
A Phase 4 clinical trial update on strategies to prevent inhibitor recurrence indefinitely in patients with Hemophilia A undergoing immune t…
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CLINICAL_TRIALS trial_result · 2023-08-29Clinical Trial Update: Phase III Expansion Trial for Determining the Safety and Efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Adolescent and Adult Patients With Hemophilia A [Phase 3]
Phase III expansion trial evaluating the safety and efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for injection in…
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CLINICAL_TRIALS trial_result · 2023-07-07Clinical Trial Update: Study to Evaluate Safety and Tolerability of a Single Dose of PF-06741086 in Chinese Adult Participants With Severe Hemophilia [Phase 1]
Phase 1 trial completed evaluating safety and tolerability of a single dose of PF-06741086 in Chinese adults with severe hemophilia.
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CLINICAL_TRIALS trial_result · 2023-06-28Clinical Trial Update: Study to Gain More Information on How Safe and Effective Jivi Works in Patients With Severe Hemophilia A (Post-marketing Investigation) [Phase 4]
Phase 4 post-marketing study updating safety and efficacy information for Jivi in patients with severe Hemophilia A.
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CLINICAL_TRIALS trial_result · 2023-05-24Clinical Trial Update: A Phase 3 Open-label Interventional Study of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein, Efanesoctocog Alfa (BIVV001), in Patients With Severe Hemophilia A [Phase 3]
Phase 3 open-label interventional study evaluating intravenous efanesoctocog alfa (BIVV001) in severe hemophilia A has completed with result…
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CLINICAL_TRIALS trial_result · 2023-05-15Clinical Trial Update: A Study of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection [Phase 2]
Phase 2 study update on repeat dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for injection in severe hemophilia …