An evaluation of the safety and efficacy of turoctocog alfa for hemophilia A

ABSTRACT

Introduction: Hemophilia A is an inherited disorder that is characterized by decreased or absent factor (F)VIII and an increased risk of bleeding. Clinical presentation of the severe form of the disease includes spontaneous bleeding into the joints and muscles, while patients with milder forms usually exhibit trauma-associated bleeding. The treatment of hemophilia aims to prevent bleeding. A number of clotting FVIII concentrates are available for managing hemophilia A, which have different safety and efficacy characteristics. Advancements in biotechnology have enabled development of recombinant factor concentrates, which thus minimize the risk of transmitting infectious diseases. Turoctocog alfa (NovoEight®, Novo Nordisk A/S, Bagsvaerd, Denmark) was the first third-generation B-domain truncated recombinant FVIII.

Areas covered: The manuscript describes the characteristics of turoctocog alfa, as well as its efficacy and safety for prophylaxis and on-demand treatment for patients with severe hemophilia A without inhibitors.

Expert opinion: In clinical trials, turoctocog alfa has demonstrated very good efficacy and safety for the prophylaxis and on-demand treatment of hemophilia A patients, as well as high hemostatic activity during surgery and in managing bleeding episodes. Post-marketing studies and real-life data are anticipated to further reinforce the value of long-term prophylaxis, and estimate the incidence of inhibitors to FVIII.

KEYWORDS:

• B-domain truncated
• factor VIII
• hemophilia A
• inhibitors
• on-demand treatment
• prophylaxis
• recombinant
• third-generation

Article highlights

• Turoctocog alfa is the first third-generation B-domain truncated recombinant FVIII.

• The pharmacokinetics of turoctocog alfa is similar to that of other available recombinant FVIII products.

• In large clinical trials, turoctocog alfa has demonstrated high efficacy and safety for prophylactic use and on-demand treatment of patients with hemophilia A.

• The incidence of inhibitors in previously untreated patients that are treated with turoctocog alfa needs further evaluation.

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 or other relationships to disclose.

Additional information

Funding

This paper was not funded.