Lucerastat is under clinical development by Idorsia Pharmaceutical and currently in Phase III for Fabry Disease. According to GlobalData, Phase III drugs for Fabry Disease have an 80% phase transition success rate (PTSR) indication benchmark for progressing into Pre-Registration. GlobalData’s report assesses how Lucerastat’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.
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GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.
Lucerastat overview
Lucerastat (ACT-434964) is under development for the treatment of Fabry disease. It is a small molecule administered through the oral route. It is an iminosugar and an analogue of miglustat. It acts by targeting glucosylceramide synthase. The drug candidate was also under development for the treatment of Sandhoff and Niemann Pick type C disease.
Idorsia Pharmaceutical overview
Idorsia Pharmaceutical (Idorsia) is a biopharmaceutical company that discovers, develops, and commercializes small molecules to treat the central nervous system and cardiovascular and immunological disorders. It is investigating daridorexant against insomnia; aprocitentan for the treatment of resistant hypertension; clazosentan to treat cerebral vasospasm associated with aneurysmal subarachnoid hemorrhage; and L-lucerastat compound targeting fabry disease. The company is also evaluating drugs against suspected acute myocardial infarction, systemic lupus erythematosus, Binge eating disorder and rare lysosomal storage disorders. It works in collaboration with Janssen Biotech to develop and commercialize aprocitentan drug candidates. Idorsia is headquartered in Allschwil, Switzerland.
For a complete picture of Lucerastat’s drug-specific PTSR and LoA scores, buy the report here.
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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.
GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.
