Half-Year 2022 Financial and Clinical Trials Update
Hemlibra
Factor VIII mimetic for treatment of hemophilia A
Indication
Hemophilia A patients
without inhibitors to factor VIII
Roche
Hemophilia A patients with and without inhibitors to Factor VIII, dosing
every 4 weeks
Phase/study
# of patients
Design
Primary endpoint
☐
Status
Phase III
HAVEN 3
N=135
Patients on FVIII episodic treatment prior to study entry:
ARM A: Hemlibra prophylaxis qw
ARM B: Hemlibra prophylaxis q2w
- ARM C: Episodic FVIII treatment; switch to Hemlibra prophylaxis possible
after 24 weeks
Patients on FVIII prophylaxis prior to study entry:
ARM D: Hemlibra prophylaxis qw
■ Number of bleeds over 24 weeks
FPI Q3 2016, recruitment completed Q2 2017
▪ Study met primary and key secondary endpoints Q4 2017
FDA granted Breakthrough Therapy Designation April 2018
Data presented at WFH 2018
Filed in US (priority review) and EU in Q2 2018
Data published in NEJM 2018; 379:811-822
Phase III
HAVEN 4
N=46
Multicenter, open-label, non-randomized study to assess the efficacy,
safety, pharmacokinetics, and pharmacodynamics of Hemlibra
administered every 4 weeks.
■ Part 1: Pharmacokinetic run-in part (N=6)
Part 2: Expansion part (N=40)
Number of bleeds over 24 weeks
FPI Q1 2017, recruitment completed Q2 2017
Pharmacokinetic run-in data at ASH 2017
Positive interim analysis outcome reported Q4 2017
Data presented at WFH 2018
Interim data filed in US and EU in Q2 2018
Data published in Lancet Haematology 2019 Jun;6(6):e295-e305
•Approved in US Q4 2018 and EU Q1 2019
CT Identifier
In collaboration with Chugai
NCT02847637
ASH-American Society of Hematology; WFH-World Federation of Hemophilia; NEJM-New England Journal of Medicine
NCT03020160
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