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Eladocagene exuparvovec - PTC Therapeutics

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Drug Profile

Eladocagene exuparvovec - PTC Therapeutics

Alternative Names: AADC deficiency gene therapy programme - PTC Therapeutics; AAV-hAADC-gene-therapy-PTC Therapeutics/National-Taiwan-University; AAV2-hAADC - PTC Therapeutics/National Taiwan University; AGIL-AADC; GT-AADC; KEBILIDI; PTC-AADC; Upstaza

Latest Information Update: 02 Dec 2024

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At a glance

  • Originator National Taiwan University
  • Developer National Taiwan University; PTC Therapeutics
  • Class Gene therapies
  • Mechanism of Action Aromatic-L-amino-acid decarboxylase replacements; Gene transference
  • Orphan Drug Status

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

    Yes - CNS disorders
  • New Molecular Entity No

Highest Development Phases

  • Registered Aromatic amino acid decarboxylase deficiency

Most Recent Events

  • 27 Nov 2024 PTC Therapeutics enters into an agreement to sell its Rare Paediatric Disease Priority Review Voucher
  • 13 Nov 2024 US FDA grants Rare Pediatric Disease Priority Review Voucher (PRV) to PTC Therapeutics for the approval of Eladocagene exuparvovec (KEBILIDITM)
  • 13 Nov 2024 Registered for Aromatic amino acid decarboxylase deficiency (In adolescents, In children, In the elderly, In infants, In adults) in USA (Intracerebral) (Accelerated approval)

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