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.
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.
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.
Report Description: The report covers in-depth analysis on Factor VIII Inhibitors Pipeline Insights, 2019. The report assesses the Factor VIII Inhibitors pipeline by stage of development (early development, pre-clinical, clinical and in approval), by application (Neurology, Immunology, Hematology, and others). In addition, the report includes key insights on other development activities, including (but not limited to) – licensing (In and Out), collaborations, acquisitions, reimbursement, patent, and regulatory designations.
The report includes in-depth company profiles of key players in Factor VIII Inhibitors Pipeline. The company profile includes key information on overview, financial highlights, product portfolio, business strategies, and key recent developments.
The report highlights information on emerging companies with potentially disruptive technologies and new market entrants.
Our research works on a holistic 360° approach to deliver high quality, validated and reliable information in our market reports. The Market estimation and forecasting involve the following steps:
Data Collation (Primary & Secondary)
In-house Estimation (Based on proprietary databases and Models)
Market-related information is assembled from both primary and secondary sources.
Top-down and bottom-up approaches: The overall market size was used in the top-down approach to estimate the sizes of other individual submarkets (mentioned in the market segmentation by product, type of manufacturing, and disease) through percentage splits from secondary and primary research. The bottom-up approach was also implemented (wherever applicable) for data extracted from secondary research to validate the market segment revenues obtained.