Factor VIII Stimulators Market: Competitive Landscape, Pipeline, and Market Analysis 2023

Blood clotting is a complex chain involving many factors that participate in various steps to form the plug. 13 cofactors are involved in the process, and Factor 8 is one of them. This clotting cascade is essential in the body. If this cascade does not work efficiently or has any factor missing or inactive, that would inhibit clot formation at the site of the injury causing continuous bleeding or a Haemorrhage. Factor 8 is responsible for converting Factor X into Factor Xa, which facilitates further conversion in the cycle. Von Willebrand factor (VWF) is another crucial glycoprotein that protects Factor VIII from proteolysis and mediates the initial contact of the platelets with the walls of the blood vessels. Thus, the deficiency of VWF in the blood plasma prevents platelet plug formation causing hemorrhagic conditions. Therefore, every component in the clotting cascade is very important for constructing platelet plugs. Factor VIII might be genetically missing or non-functional in some individuals. This disease is called Hemophilia A. This disease prevents blood clotting and might be fatal if external medication is not provided on time. Extensive research is being conducted globally to discover new and novel molecules to be used as substitutes for Factor VIII to control bleeding in people with Hemophilia. The development and launch of new products will thus increase the market’s revenue in the forecasted years.  According to the U.S. Department of Health and Human Services, Hemophilia occurs in approximately 1 in every 5617 male births in the USA, with more than half diagnosed with a severe form of Hemophilia A and 400 boys born with Hemophilia A yearly.

A number of these inhibitors are in the market, while many are in ongoing clinical trials. Many companies have various products in their pipelines, with 48 products in different stages of clinical trials. 21 products in Phase 4, 10 in Phase 3, 9 in Phase 2, and 4 in Phase 1 of the clinical trials.

Key Developments of Factor VIII Stimulators

• In February 2023, FDA approved once-weekly ALTUVIIIO, which is a new class of Factor VIII therapy for Haemophilia A.
• In May 2022, FDA granted ALTUVIIIO Breakthrough Therapy designation for treating Haemophilia A.
• In February 2021, FDA granted ALTUVIIIO Fast Track designation for treating Haemophilia A.

Approved molecules of Factor VIII Stimulators

• Advate (Octocog Alfa)
• Xyntha (Moroctocog Alfa)
• Kogenate FS (Octocog Alfa Sucrose Formulation)
• Eloctate (Efraloctocog Alfa)
• Novoeight (Turoctocog Alfa)
• Adynovate (Rurioctocog Alfa Pegol)
• Nuwiq (Simoctocog Alfa)
• Jivi (Damoctocog Alfa Pegol)
• Wilate (Human Factor VIII/Von Willebrand Factor)
• Kovaltry (Octocog Alfa)
• Biostate (Factor VIII/Von Willebrand Factor)
• Haemate-P (Human Plasma Derived Von Willebrand Factor/FVIII Complex)
• Optivate (Factor VIII/Von Willebrand Factor)
• Octanate (Human Factor VIII/Von Willebrand Factor)
• Alphanate (Human Plasma Derived Von Willebrand Factor/FVIII Complex)
• Abstilla (Lonoctocog Alfa)
• Ancain (Omfiloctocog Alfa)
• Bioclate (Recombinant Factor VIII)
• Aafact (Monoclonal Purified Factor VIII)
• Fanhdi (Human Plasma Derived Coagulation Factor VIII)
• Hemofil-M (Human Plasma Derived Coagulation Factor VIII)

Drugs in the Pipeline of Factor VIII Stimulators

• Esperoct (Turoctocog Alfa Pegol)
• Mim8
• Obizur (Susoctocog Alfa)
• Altuviiio (Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-Ehtl)
• Roctavian (Valoctocogene Roxaparvovec)
• FRSW107
• Greengene F (Beroctocog Alfa)
• Kogenate FS Liposome (Octocog Alfa (Pegylated))
• Dirloctocogene Samoparvovec (RG6357)
• Giroctocogene Fitelparvovec (SB-525)
• ET3i
• Haemoctin (Human Plasma Derived Coagulation Factor VIII)
• Koate –DVI (Double Viral Inactivation Solvent Treated Human Factor VIII)
• TAK-754
• Peg Recombinant Human Coagulation Factor VIII-Fc Fusion Protein (FRSW117)
• ASC-618
• BAX 826
• Beriate P (Human Plasma Derived Coagulation Factor VIII)
• Immunate SD (Human Plasma Derived Coagulation Factor VIII)
• Longate (Plasma-Derived Liposomal Factor VIII)
• RG6358
• SIG-001
• YUVA-GT-F801
• Glycopegylated Turoctocog Alfa Pegol (NN7170)
• Peboctocogene Camaparvovec (DTX201)
• Pegylated Liposome Simoctocog Alfa (PBB 8 IN)
• Simoctocog Alpha/Von Willebrand Factor (OCTA101)

Clinical Activity and Developments of Factor VIII Stimulators

As of June 2023, 33 companies have approximately 48 products for 47 diseases. For these diseases, 93 trials are being conducted by players globally. For instance,

• Takeda is conducting a Phase 4 clinical trial to study the safety of Advate in Haemophilia A patients in India, which is expected to complete by October 2023.
• Swedish Orphan Biovitrum is conducting a Phase 4 clinical trial to assess joint health in Haemophilia A and B patients on prophylaxis with Efmoroctocog Alfa ro Eftrenonacog Alfa that is expected to complete by December 2025.
• Grifols Biologicals, LLC. is conducting a Phase 4 clinical trial to study the efficacy of Alphanate FVIII/ Von Willebrand Factor concentrate in Type 3 Von Willebrand patients that is expected to complete by March 2029.