Sinbaglustat is under clinical development by Idorsia Pharmaceutical and currently in Phase I for Tay-Sachs Disease. According to GlobalData, Phase I drugs for Tay-Sachs Disease does not have sufficient historical data to build an indication benchmark PTSR for Phase I. GlobalData uses proprietary data and analytics to create drugs-specific PTSR and LoA in the Sinbaglustat LoA Report. Buy the report here.

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.

Sinbaglustat overview

Sinbaglustat (ACT-519276) is under development for the treatment of Tay-Sachs disease or GM2 gangliosidosis, a rare lysosomal storage disorders (LSD). The drug candidate acts by targeting glucosylceramide synthase and beta glucocerebrosidase. It is administered through oral route.

It was also under development for the treatment of central nervous system disorders.

Idorsia Pharmaceutical overview

Idorsia Pharmaceutical (Idorsia), a subsidiary of Idorsia Ltd, is a biopharmaceutical company that discovers, develops, and commercializes small molecules. The company’s portfolio products include daridorexant, aprocitentan, lucerastat, sinbaglustat, ACT-777991, ACT-1014-6470, IDOR-1117-2520 and others. Its products treat therapeutic areas such as central nervous system (CNS), cardiovascular, immunological disorders and orphan diseases. Idorsia collaborates with Janssen Biotech to develop and commercialize aprocitentan drug candidates. The company operates through offices in the Netherlands and Switzerland. Idorsia is headquartered in Allschwil, Basel-Landschaft, Switzerland.

For a complete picture of Sinbaglustat’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.