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
HemophiliaView entire presentation