The study analyzed that the lysosomal storage disorder therapeutics pipeline comprises approximately 74 drug candidates, of which one drug candidate is in Filed stage, six are in Phase III stage, seven are in Phase II stage, 16 are in Phase I stage, 33 are in Pre-Clinical stage and 11 are in the Discovery stage of development. Lysosomal storage disorder is defined as a heterogeneous group of rare inherited disorders characterized by various enzyme deficiencies, that ultimately results in cellular dysfunction and clinical abnormalities.
According to the research findings, around 23.0% of drug candidates for lysosomal storage disorder pipeline are being developed to be administered by intravenous route, 11.0% by oral route and 12.0% are to be administered by other routes, that include intracerebral, intracerebroventricular and intrathecal. Route of administration of around 54.0% drug candidates of lysosomal storage disorder pipeline has not been disclosed.
Many companies are involved in the development of drugs for the lysosomal storage disorder therapeutics pipeline, with their products in different phases. Ultragenyx Pharmaceutical Inc. has its drug candidate in Filed stage while GlaxoSmithKline plc has its drug candidate in the Phase III stage of development. Companies such as Sanofi Genzyme, Eloxx Pharmaceuticals, Inc. and Lysogene S.A. have their drug candidates in the Phase II stage of development. ArmaGen, Inc. is in the process of developing four drug candidates of which one is in the Phase II stage of development, and the other drug is in the Phase I stage of development and two are in the Pre-Clinical stage of development.
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Some of the other key players developing drugs for the treatment of lysosome storage disorder include GlaxoSmithKline plc, Sanofi Genzyme, Amicus Therapeutics, Inc., Ultragenyx Pharmaceutical Inc., Orphazyme ApS, ArmaGen, Inc., Eloxx Pharmaceuticals, Inc., Lysogene S.A., Sucampo Pharmaceuticals, Inc., Abeona Therapeutics Inc. and Esteve S.A.
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