Niemann-Pick Disease is an indication for drug development with over 30 pipeline drugs currently active. According to GlobalData, preregistered drugs for Niemann-Pick Disease have a 50% 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 Niemann-Pick Disease 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.
Niemann-Pick Disease overview
Acid sphingomyelinase deficiency (ASMD) is a rare genetic disorder that affects the metabolism of a lipid called sphingomyelin. ASMD is caused by mutations in the SMPD1 gene, which encodes the enzyme acid sphingomyelinase (ASM). ASM is responsible for breaking down sphingomyelin in the lysosomes of cells. When ASM is deficient, sphingomyelin accumulates in various tissues and organs, causing damage and dysfunction. ASMD is also known as Niemann-Pick disease types A and B, depending on the severity and presence of neurological symptoms. ASMD can cause hepatosplenomegaly, thrombocytopenia, ataxia, dystonia, dementia, and bone abnormalities. ASMD is inherited in an autosomal recessive manner and has no cure. Treatment is supportive and symptomatic.
For a complete picture of PTSR and LoA scores for drugs in Niemann-Pick Disease, 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.
