Mutational Disorders is an indication for drug development with over 190 pipeline drugs currently active. According to GlobalData, preregistered drugs for Mutational Disorders 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 Mutational Disorders 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.

Mutational Disorders overview

Mutational disorders, alternatively termed genetic or genomic disorders, stem from modifications in an individual’s DNA sequence. These alterations, encompassing substitutions, deletions, insertions, or rearrangements of genetic material, can impact a single gene or multiple genes in both somatic and germ cells, resulting in inherited or sporadic disorders. The classification of mutational disorders spans various categories. Single gene disorders, such as cystic fibrosis, sickle cell anemia, and Huntington’s disease, arise from mutations in a solitary gene, often following Mendelian inheritance patterns. Chromosomal disorders, like Down syndrome, Turner syndrome, and Klinefelter syndrome, result from structural or numerical abnormalities in chromosomes. Polygenic disorders involve the interplay of multiple genes and environmental factors, encompassing prevalent conditions like diabetes, heart disease, and certain cancers. Mitochondrial disorders, linked to mutations in mitochondrial DNA affecting energy production, include examples such as Leigh syndrome and mitochondrial myopathy.

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