Hemophilia Therapeutics Pipeline Analysis Overview
The pipeline analysis based on route of administration of active drug candidates showed, that around most of the hemophilia drug candidates are being developed to be administered by intravenous route.
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Most of the pharma and biotech companies are developing the therapeutics for hemophilia in collaboration with educational institutes. In September 2016, Roche in collaboration with Chugai Pharmaceutical Co. Ltd. initiated a Phase III trial to evaluate the safety, pharmacokinetics and efficacy of prophylactic subcutaneous emicizumab in patients (aged 12 years) suffering from hemophilia A without factor VIII inhibitors.
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Many drug candidates received USFDA designations namely Orphan, Fast Track or Breakthrough designations in their clinical stages for the treatment of hemophilia. In August 2016, Orphan Drug Designation was granted to Phase III drug candidate of Chugai Pharmaceuticals, by Ministry of Health, Labour and Welfare, Japan.
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Some of the key players developing drugs for the treatment of hemophilia include Sangamo BioSciences, Inc., Caisson Biotech, Inc., XL-protein GmbH, and others.
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