Sepiapterin is under clinical development by PTC Therapeutics and currently in Phase III for Phenylketonuria (PKU). According to GlobalData, Phase III drugs for Phenylketonuria (PKU) have a 100% phase transition success rate (PTSR) indication benchmark for progressing into Pre-Registration. GlobalData’s report assesses how Sepiapterin’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication 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.
Sepiapterin overview
Sepiapterin (PTC-923 (CNSA-001)) is under development for the treatment of moderate to severe diabetic gastroparesis, tetrahydrobiopterin (BH4) deficiency resulting in hyperphenylalaninemia and phenylketonuria, segawa disease, central nervous system disorders and unspecified indications. The drug candidate is a tetrahydrobiopterin derivative. It is a small molecule administered through oral route. The drug candidate acts by targeting tetrahydrobiopterin.
it was also under development for the treatment of Parkinson's disease.
PTC Therapeutics overview
PTC Therapeutics discovers, develops, and markets medicines for the treatment of rare disorders. Its marketed products include Evrysdi (risdiplam) for spinal muscular atrophy, Translarna (ataluren), and Emflaza (deflazacort) for the treatment of Duchenne muscular dystrophy. The company’s pipeline candidates target the treatment of Huntington’s disease; children with inherited mitochondrial disease; leiomyosarcoma; acute myelogenous leukemia; and aromatic L-amino acid decarboxylase deficiency. PTC Therapeutics works in collaboration with various biotechnology and pharmaceutical companies for advancing its pipeline candidates. It operates through its subsidiaries in the US and Europe. PTC Therapeutics is headquartered in South Plainfield, New Jersey, the US.
For a complete picture of Sepiapterin’s drug-specific PTSR and LoA scores, 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.
