Sphingolipidoses is an indication for drug development with over 130 pipeline drugs currently active. According to GlobalData, preregistered drugs for Sphingolipidoses have a 75% likelihood of approval (LoA) indication benchmark. GlobalData’s report assesses how phase transition success rate (PTSR) and likelihood of approval (LoA) scores for pipeline drugs in Sphingolipidoses compared to historical 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.

Sphingolipidoses overview

Sphingolipidoses are a heterogeneous group of inherited disorders of lysosomal storage diseases affecting primarily the central nervous system. It is characterized by the harmful accumulation of glycosphingolipids and phosphosphingolipids. Fabry disease, Gaucher disease, Krabbe disease, metachromatic leukodystrophy Niemann-pick syndrome, and Tay-Sachs disease are examples of sphingolipidoses. Most of them are autosomal recessive. Based on family history, enzymatic assays, and mutational analysis are diagnostic features. Gene therapy and enzyme replacement therapy are treatment options.

For a complete picture of PTSR and LoA scores for drugs in Sphingolipidoses, 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.