Lysosomal Storage Disorder is an indication for drug development with over 250 pipeline drugs currently active. According to GlobalData, preregistered drugs for Lysosomal Storage Disorder have a 80% 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 Lysosomal Storage Disorder 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.
Lysosomal Storage Disorder overview
Lysosomal Storage Disorder is a rare genetic disease. Defective functioning of lysosomes causes the accumulation of substrates in various organs. It is predominant during pregnancy or right after birth. Lipidoses, Mucopolysaccharidoses, Sphingolipidoses are common forms of LSD. No treatment for LSD. Enzyme replacement therapies and stem cell therapies are commonly used therapies for LSD.
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For a complete picture of PTSR and LoA scores for drugs in Lysosomal Storage Disorder, 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.
