The hemophilia therapeutics pipeline is expected to grow in future due to rise in prevalence of genetic variation. Hemophilia which is symptomatized by excessive bleeding in brains, joints, nose and mouth. Some of the drivers for the growth of the hemophilia therapeutics pipeline include increasing incidence of hemophilia, less number of drugs present for treatment of hemophilia.
Hemophilia is the disease, mostly inherited, in which people have little or no clotting factor. Clotting factor is required for normal blood clotting. These proteins work with platelets to promote the clotting of the blood. There are two types of hemophilia. Hemophilia A, in which clotting factor VIII is missing or present in very low level. Hemophilia B, in which clotting factor IX is missing or present in very low levels.
Most of the large pharma companies are spending in research and development for treatment of haemophilia, which are in clinical trials. Pfizer, Inc. is developing a gene therapy in collaboration with Spark Therapeutics, Inc., through SPK-FIX program for treatment of haemophilia, as a potential onetime therapy. SPK-9001 is a bio-engineered adeno-associated virus (AAV) capsid expressing a codon-optimized, high-activity human factor IX variant, which was advanced using Spark’s proprietary technology. Chugai Pharmaceutical is developing a compound in collaboration with F. Hoffmann-La Roche AG, Emicizumab, which is considered as a Breakthrough Therapy by the US Food and Drug Administration. LFB USA, Inc. is introducing a biological, wich is recombinant Coagulation FVIIa for the treatment of haemophilia. Coagulation FVIIa is currently in Phase III clinical development stage. Alnylam Pharmaceuticals Inc. is developing a drug for the treatment of haemophilia, ALN-AT3SC, which lowers the level of plasma antithrombin. ALN-AT3SC drug led to restoration of homeostasis in people with haemophilia. OPKO Biologics Ltd. Is developing a drug MOD-5014, whih is a long-acting recombinant FVIIa for the treatment of hemophiia A or B. Octapharma AG is developing a compound fr the treatment of haemophilia, Wilate, which is stable, double virus inactivated, stable, highly purified concentrate of freeze-dried, active von Willebrand factor (VWF) and FVIII. Wilate is currently in Phase III clinical development. Novo Nordisk A/S is estimating a homeostatic effect of turoctocog alfa pegol, which is currently in Phase III. Bayer AG is developing a compound BAY1093884. BAY1093884 is an anti-TFPI antibody which is currently in Phase I. Shire Plc is developing a compound SHP656, which is currently in Phase I clinical development.
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Some of the key players having a pipeline of hemophilia therapeutics include Shire Plc, Biogen, Pfizer Inc., Novo Nordisk A/S, CSL Behring, F. Hoffmann-La Roche Ltd., Bayer AG, Biotest AG, Kedrion Biopharma Inc., and Octapharma AG.
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