Roche Pharmaceutical Development and Sales Overview slide image

Roche Pharmaceutical Development and Sales Overview

Hemlibra (emicizumab, RG6013) Factor VIII mimetic for treatment of hemophilia A Indication Hemophilia A patients with and without inhibitors to Factor VIII Hemophilia A mild to moderate patients without inhibitors to Factor VIII Phase/study Phase III HAVEN 5 Phase III HAVEN 6 Roche # of patients Design N=85 Patients with Hemophilia regardless of FVIII inhibitor status on prophylactic or episodic treatment prior to study entry: ā˜ ARM A: Hemlibra prophylaxis qw ARM B: Hemlibra prophylaxis q4w ARM C: No prophylaxis (control arm) Number of bleeds over 24 weeks N=70 Multicenter, open-label study to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of Hemlibra in patients with mild or moderate Hemophilia A without FVIII inhibitors Hemlibra qw (1.5mg/kg), q2w (3.0mg/kg) or q4w (6.0mg/kg) (patients preference) Safety and efficacy Primary endpoint Status FPI Q2 2018 Recruitment completed Q1 2019 Filed in China Q2 2020 Approved in China Q2 2021 CT Identifier NCT03315455 In collaboration with Chugai ASH-American Society of Hematology; ISTH=International Society on Thrombosis and Haemostasis ā˜ FPI Q1 2020 Recruitment completed Q1 2021 Interim data presented at ASH 2021 and primary data presented at ISTH 2022 Filed in EU Q4 2021 NCT04158648 61 Hemophilia
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