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A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion (NTUH-AADC-011)

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Trial Profile

A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion (NTUH-AADC-011)

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 18 May 2024

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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

  • 14 May 2024 According to a ClearPoint Neuro media release, announced today that FDA has accepted for filing the Biologics License Application (BLA) for Upstaza (eladocagene exuparvovec), a gene therapy for the treatment of AADC deficiency. The application has been granted Priority Review with a target regulatory action date of Nov 13, 2024 - 14 may.
  • 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.
  • 27 Apr 2023 Results assessing the efficacy and safety of eladocagene exuparvovec (Data were extracted on January 4, 2022) in patients with aromatic l-amino acid decarboxylase deficiency in 3 studies (AADC-CU/1601 [n=8], AADC-010 [n=10], and AADC-011 [n=12] in patients aged 18-102 months, presented at the 75th Annual Meeting of the American Academy of Neurology 2023.

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