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PTC FA

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Drug Profile

PTC FA

Alternative Names: AGIL FA; GT-FA; PTC-FA

Latest Information Update: 30 May 2023

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At a glance

  • Originator Agilis Biotherapeutics
  • Developer PTC Therapeutics
  • Class Gene therapies
  • Mechanism of Action Frataxin expression stimulants; Gene transference
  • Orphan Drug Status

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

    Yes - Friedreich's ataxia
  • New Molecular Entity No

Highest Development Phases

  • Discontinued Friedreich's ataxia

Most Recent Events

  • 23 May 2023 Discontinued - Preclinical for Friedreich's ataxia in USA (unspecified route)
  • 16 Jan 2023 Preclinical development is ongoing in USA (PTC Therapeutics website, January 2023)
  • 28 May 2022 No recent reports of development identified for preclinical development in Friedreich's ataxia in USA

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