rFVIIIFC for hemophilia A prophylaxis

ABSTRACT

Introduction: rFVIIIFC was the first extended half-life product to complete the phase 3 development program and be registered. It was developed to reduce the high treatment burden imposed by prophylaxis. It is now one of four extended half-life products available for a variety of indications in hemophilia A. This article focus on the efficacy use of rFVIIIFC in the prevention of bleeds in hemophilia A.

Areas covered: This article provides an update on efficacy data from three clinical studies describing the use of rFVIIIFC in the treatment and prevention of bleeds in hemophilia A. The update includes the efficacy use of rFVIII in all age groups, in the perisurgical setting, in immune tolerance induction, and in improving the quality of life of patients. The role of rFVIIIFC prophylaxis in the face of rapidly evolving non-replacement therapy and gene therapy is summarized.

Expert commentary: The role of rFVIIIFC in hemophilia A prophylaxis is uncertain in the light of development of newer prophylaxis agents with better route of administration, improved pharmacokinetic and superior efficacy profiles. While rFVIIIFC was primarily developed for prophylaxis in hemophilia A, this role may change in the face of competitive extended half-life products and non-replacement therapies.

KEYWORDS:

• Efficacy
• hemophilia A
• prophylaxis
• non-replacement therapies
• rFVIIIFC

Declaration of interest

The authors have no 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. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

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

Additional information

Funding

This paper was not funded.