Half-Year 2022 Financial and Clinical Trials Update
Hemlibra
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
# of patients
Phase III
HAVEN 5
N=85
Phase III
HAVEN 6
N=70
Roche
•
Design
.
Patients with Hemophilia regardless of FVIII inhibitor status on
prophylactic or episodic treatment prior to study entry:
.
Arm A: Hemlibra prophylaxis qw
Arm B: Hemlibra prophylaxis q4w
• Arm C: No prophylaxis (control arm)
Number of bleeds over 24 weeks
Primary endpoint
Status
■ FPI Q2 2018
Recruitment completed Q1 2019
Filed in China Q2 2020
▪ Approved in China Q2 2021
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
■ 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
CT Identifier
NCT03315455
In collaboration with Chugai
ASH-American Society of Hematology; ISTH=International Society on Thrombosis and Haemostasis
NCT04158648
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HemophiliaView entire presentation