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A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC

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Trial Profile

A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 04 Mar 2025

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At a glance

  • Drugs Eladocagene exuparvovec (Primary)
  • Indications Aromatic amino acid decarboxylase deficiency; Inborn error amino acid metabolic disorders
  • Focus Registrational; Therapeutic Use

Most Recent Events

  • 27 Feb 2025 According to a PTC Therapeutics media release, eladocagene exuparvovec-tneq for the treatment of AADC deficiency in the U.S., approved in November 2024.
  • 08 Aug 2024 According to a PTC Therapeutics media release, the BLA was accepted by FDA with priority review; the target regulatory action date is November 13, 2024.
  • 19 Mar 2024 According to a ClearPoint Neuro media release, the PTC Therapeutics has completed its BLA submission to the U.S. Food and Drug Administration (FDA) for the approval of Upstaza (eladocagene exuparvovec), an investigational treatment for AADC Deficiency.

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