Factor VIII Inhibitors, Potential Market Opportunity & Clinical Pipeline Insights, 2019

Factor VIII was also known, as an antihemophilic factor is referred as essential blood-clotting protein. Factor VIII is said to be produced outside the liver in liver sinusoidal cells and endothelial cells of the body. It is mixed in the bloodstream in an inactive form and binds to another molecule (von Willebrand factor) until the occurrence of damage to blood vessels. By injury, coagulation factor VIII responses by getting activated and it separates from von Willebrand factor. The formation of an FVIII inhibitor is said to be a T-cell dependent event, which involves antigen-presenting cells, T- and B-helper lymphocytes. The antibodies in inhibitor patients can simultaneously target multiple FVIII epitopes, and these epitope targeting will change over time. FVIII inhibitors are classified on the basis of kinetics and extent of inhibition into Type I inhibitors, which are more common in severe hemophilia and Type II inhibitors, which are common in inhibitor patients having mild hemophilia or in patients without hemophilia and developed an acquired FVIII inhibitor.

Market Overview

FVIII inhibitors interfere with the infused factor concentrates, thereby making them ineffective and inevitably using the most costly and less effective alternative hemostatic agents.  Currently, inhibitor development comes under the most significant treatment complication in hemophilia patients, which is associated with substantial morbidity and with decreased quality of life. Thus, FVIII inhibitor development results from a complex interaction existing between the patient’s immune system, environmental and genetic risk factors. The main purpose of the treatment is the eradication of the inhibitor by immune tolerance.

Several studies are under different stages of drug development in clinical trials of factor VIII inhibitors, which are majorly applicable for treating hemophilia condition. Among them, the studies in phase 3 stage of drug development includes drugs like turoctocog alfa, BAX802, Emicizumab, Fitusiran (ALN-AT3SC), Rituximab, Cyclophosphamide that is going very vast that will add to the market growth and only a few numbers of studies are under phase 2, phase 1, and early phases of drug development including drugs like Bortezomib, BAY1093884, Concizumab, MOD-5014. Thus further factor VIII inhibitors have to be designed to increase the market growth.

The major factors that is responsible for the market segment’s growth is the increasing incidence of hemophilia A, the increase in adoption of prophylactic treatment, and the development of novel drugs with extended action. Additionally, increase in demand for novel recombinant products with extended half-life, introducing plasma-derived products for recombinant factor VIII products at lower costs, and the appearance of monoclonal antibodies and gene therapy drug products are further driving the growth of the factor VIII inhibitors market.

Key Developments

• In Feb 2019, the U.S. FDA has approved Cablivi (caplacizumab) in combination with plasma exchange and immunosuppression for treating acquired thrombotic thrombocytopenic purpura (aTTP) in adults. Cablivi is the first FDA approved therapy specifically indicated for the treatment of aTTP.


• In Feb 2019, CHMP recommends EU approval of Roche's Hemlibra ® (emicizumab) for people with severe haemophilia A without factor VIII inhibitors.

Segmentation

This pipeline analysis report segments the Factor VIII inhibitors based on therapies employed (monotherapy and undisclosed), RoA (oral and undisclosed), therapeutic modalities (small molecules and disclosed), drugs under development (discovery, pre-clinical, phase I, phase I/II, phase II, and phase III), and recruitment status (recruiting, active not recruiting, enrolling by invitation, and undisclosed).

By Application: Neurology, Immunology, Hematology, Critical Care, Pulmonology, Hemato-Oncology, and Rheumatology.

The key players involved in the development of Factor VIII Inhibitors are Bayer, Bioverativ therapeutics, Baxalta Innovations, Chugai Pharmaceuticals, CSL Behring, Hoffmann-La Roche, Novo Nordisk, Octapharma, and the Shire.