Hemophilia
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Sign up freeTop drugs studied for this disease
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FDA approval · 2022-06-06FDA Approved: HEMLIBRA — Supplemental Application (Genentech, Inc.)
FDA approved a supplemental application for Hemlibra (emicizumab) submitted by Genentech, Inc. The action updates the product’s labeling and…
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CLINICAL_TRIALS trial_result · 2022-05-19Clinical Trial Update: A Phase 1/2 Study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects [Phase 1, Phase 2]
Phase 1/2 trial of SHP648, an AAV-based gene transfer for Hemophilia B, is terminated with results posted.
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CLINICAL_TRIALS trial_result · 2022-03-09Clinical Trial Update: A Study to Evaluate the Safest Dose Range for FEIBA in Hemophilia A Patients With Inhibitors on Emicizumab [Phase 4]
A Phase 4 clinical trial to evaluate the safest dose range for FEIBA in hemophilia A patients with inhibitors who are on emicizumab.
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CLINICAL_TRIALS trial_result · 2022-02-04Clinical Trial Update: A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors [Phase 3]
Phase 3 trial of Fitusiran in severe Hemophilia A and B patients without inhibitors completed; results posted in 2022.
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CLINICAL_TRIALS trial_result · 2021-12-21Clinical Trial Update: A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors [Phase 3]
Phase 3 trial update for fitusiran (ALN-AT3SC) in severe Hemophilia A and B patients with inhibitors has completed with results posted.
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CLINICAL_TRIALS trial_result · 2021-11-30Clinical Trial Update: Study of rFVIIIFc for Immune Tolerance Induction (ITI) in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies [Phase 4]
A Phase 4 clinical trial evaluating rFVIIIFc for immune tolerance induction in hemophilia A patients with inhibitors who previously failed I…
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CLINICAL_TRIALS trial_result · 2021-11-23Clinical Trial Update: Alphanate in Immune Tolerance Induction Therapy [Phase 2]
Phase 2 trial update for Alphanate in immune tolerance induction therapy was terminated; results posted.
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CLINICAL_TRIALS trial_result · 2021-11-11Clinical Trial Update: BT200 in Hereditary Bleeding Disorders [Phase 2]
Phase 2 trial update for BT200 in hereditary bleeding disorders has completed, investigating von Willebrand disease and Hemophilia A.
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CLINICAL_TRIALS trial_result · 2021-10-27Clinical Trial Update: An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A [Phase 3]
Open-label Phase 3 trial evaluating safety and efficacy of recombinant Factor VIII in adults with severe Hemophilia A; study completed with …
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CLINICAL_TRIALS trial_result · 2021-10-20Clinical Trial Update: BAX 802 in CHA With Inhibitors [Phase 3]
Phase 3 trial of BAX 802 in Hemophilia A was terminated.
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CLINICAL_TRIALS trial_result · 2021-10-08Clinical Trial Update: Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg [Phase 3]
A single-arm Phase 3 trial evaluating valoctocogene roxaparvovec at 4e13 vg/kg in Hemophilia A has completed and posted results.
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CLINICAL_TRIALS trial_result · 2021-09-28Clinical Trial Update: Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of NUWIQ
Non-interventional post-authorisation study assessing immunogenicity, safety, and efficacy of NUWIQ in Hemophilia A has completed with resul…
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CLINICAL_TRIALS trial_result · 2021-09-23Clinical Trial Update: Study of Coagulation Factor VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia A and B [Phase 2]
Phase 2 clinical trial updating coagulation factor VIIa variant marzeptacog alfa (activated) in adults with hemophilia A or B with inhibitor…
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FDA approval · 2021-09-02FDA Approved: HEMLIBRA — Supplemental Application (Genentech, Inc.)
FDA approved a supplemental application for Hemlibra (emicizumab) from Genentech, expanding or updating its label.
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CLINICAL_TRIALS trial_result · 2021-08-24Clinical Trial Update: Study of Pharmacokinetics, Efficacy and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients - A Continuation of Clinical Study 069901 [Phase 2, Phase 3]
Clinical trial update on pharmacokinetics, efficacy, and safety of recombinant and protein-free Factor VIII (rAHF-PFM) in Hemophilia A patie…
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CLINICAL_TRIALS research · 2021-08-24Clinical Trial Update: Safety and Efficacy Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients Undergoing Surgery [Phase 2, Phase 3]
A Phase 2/Phase 3 clinical trial evaluating the safety and efficacy of a recombinant and protein-free Factor VIII (rAHF-PFM) in Hemophilia A…
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CLINICAL_TRIALS trial_result · 2021-08-09Clinical Trial Update: Study of Next-Generation Recombinant Factor IX Variant in Adult Subjects With Hemophilia B [Phase 2]
Phase 2 trial update on a next-generation recombinant Factor IX variant for Hemophilia B; results posted after completion.
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CLINICAL_TRIALS trial_result · 2021-07-30Clinical Trial Update: Nonacog Alfa Prophylaxis And Treatment Of Bleeding Episodes In Previously Treated Patients With Hemophilia B [Phase 4]
Phase 4 trial update evaluating nonacog alfa for prophylaxis and treatment of bleeding episodes in previously treated patients with Hemophil…
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CLINICAL_TRIALS trial_result · 2021-07-27Clinical Trial Update: PF-06741086 Long-term Treatment in Severe Hemophilia [Phase 2]
Phase 2 long-term treatment trial for PF-06741086 in severe Hemophilia A or B has completed with results posted.
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CLINICAL_TRIALS trial_result · 2021-07-16Clinical Trial Update: Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia [Phase 1]
Phase 1 trial update for marzeptacog alfa (activated), a coagulation factor VIIa variant, in adults with hemophilia showing completed status…
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CLINICAL_TRIALS trial_result · 2021-06-11Clinical Trial Update: A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors [Phase 3]
Phase 3 trial evaluating safety and tolerability of prophylactic emicizumab in Hemophilia A patients with inhibitors has completed with resu…
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EMA approval · 2021-05-19EMA Authorised: NovoEight (turoctocog alfa) — Novo Nordisk A/S
NovoEight (turoctocog alfa) by Novo Nordisk A/S has been authorised for use in Hemophilia A.
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CLINICAL_TRIALS trial_result · 2021-05-05Clinical Trial Update: Efficacy and Safety Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients in Canada With Hemophilia A - A Continuation of Baxter Study 060101 [Phase 2, Phase 3]
A continuation study evaluating recombinant and protein-free Factor VIII (rAHF-PFM) in pediatric Hemophilia A patients conducted in Canada; …
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CLINICAL_TRIALS trial_result · 2021-05-03Clinical Trial Update: Moroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Hemophilia A Patients [Phase 4]
Phase 4 trial update for Moroctocog Alfa (AF-CC) in prophylaxis and treatment of bleeding episodes in previously treated Hemophilia A patien…
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CLINICAL_TRIALS trial_result · 2021-05-03Clinical Trial Update: China ADVATE PTP Study [Phase 4]
Phase 4 clinical trial update for ADVATE in China, completed study under Baxalta/Shire sponsorship for Hemophilia A.