Emicizumab for the prevention of bleeds in hemophilia A

ABSTRACT

Introduction: The management of hemophilia A with and without inhibitors is challenging with high treatment burden of prophylactic regimens, musculoskeletal complications, poor treatment compliance, poor venous access and therapies with suboptimal levels.

Areas covered: Emicizumab is a bispecific monoclonal antibody recently approved for prevention of bleeds in hemophilia A patients with and without inhibitors. This review is a synthesis of several recently completed emicizumab clinical trials with the emphasis of its mechanism of action, efficacy, safety, and pharmacokinetic profile when used for prevention of bleeds in hemophilia A patients of all age groups with and without inhibitors. We also review the role of emicizumab in the era of rapidly evolving novel therapies in hemophilia A.

Expert opinion: Data from completed clinical studies indicate that emicizumab is poised to address current unmet needs in hemophilia A. It has high efficacy in the prevention of bleeds and a favorable safety profile. Subcutaneous dosing and versatile dosing regimens make emicizumab an ideal drug to address current unmet needs in hemophilia A management across all age groups. How emicizumab fits in the current rapidly evolving hemophilia A therapeutic landscape will be exciting to watch in the coming period.

KEYWORDS:

• Emicizumab
• hemophilia A
• prophylaxis
• bispecific monoclonal antibody
• clinical trials
• Haven study
• safety
• efficacy
• pharmacokinetic

Declaration of interest

The author is a clinical trialist who has received research funding, advisory board fees and speaker fees from Alnylam, Amgen, Bayer, Biogen, Biotest, Baxalta, CSL, Catalyst Bioscience, Novo, Pfizer, Roche and Sobi. The author has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial relationships or otherwise to disclose.

Additional information

Funding

This paper was not funded.