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Sepiapterin - PTC Therapeutics

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Drug Profile

Sepiapterin - PTC Therapeutics

Alternative Names: CNSA 001; PTC-923; Sephience

Latest Information Update: 26 Jun 2025

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At a glance

  • Originator Censa Pharmaceuticals
  • Developer Censa Pharmaceuticals; PTC Therapeutics
  • Class Amines; Gastrokinetics; Ketones; Pteridines; Small molecules; Urologics
  • Mechanism of Action Phenylalanine hydroxylase stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Phenylketonuria
  • New Molecular Entity Yes

Highest Development Phases

  • Registered Phenylketonuria
  • Preclinical Lupus nephritis
  • Discontinued Diabetic gastroparesis; Dystonia

Most Recent Events

  • 26 Jun 2025 Discontinued - Phase-I for Dystonia (In volunteers) in Australia (PO) (PTC Therapeutics pipeline, June 2025)
  • 26 Jun 2025 Discontinued - Phase-II for Diabetic gastroparesis in USA (PO) (PTC Therapeutics pipeline, June 2025)
  • 23 Jun 2025 Registered for Phenylketonuria (In children, In adolescents, In adults, In the elderly) in Liechtenstein, Norway, Iceland, European Union (PO) - First global approval

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