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.

Lipid Storage Disorders (Lipidoses) overview

Lipid storage disorders, or lipidoses, encompass inherited metabolic conditions where fats accumulate due to enzyme deficiencies, impacting various organs. Gaucher, Niemann-Pick, Tay-Sachs, Fabry, and Krabbe diseases are examples. Symptoms, varying by disorder, may include neurological problems, organ enlargement, and skeletal abnormalities. Treatments focus on symptom management, employing enzyme replacement, substrate reduction therapies, and supportive care. Managing these complex conditions requires tailored approaches; however, some lack specific treatments. Genetic counseling aids in understanding inheritance patterns and risks for affected families, offering guidance amidst these rare and diverse disorders.

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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.