Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?

Eladocagene exuparvovec - PTC Therapeutics

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; PTC-AADC; Upstaza

Latest Information Update: 22 Mar 2024

Price : $50 *
  • Adis is an information provider. We do not sell or distribute actual drugs.
  • Final gross price and currency may vary according to local VAT and billing address.
  • Your purchase entitles you to full access to the information contained in our drug profile at the time of purchase.
  • A link to download a PDF version of the drug profile will be included in your email receipt.

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

  • 19 Mar 2024 Preregistration for Aromatic amino acid decarboxylase deficiency (In infants, In children, In adolescents, In adults, In the elderly) in USA (Intracerebral)
  • 19 Mar 2024 Registered for Aromatic amino acid decarboxylase deficiency (In children, In infants, In adults, In adolescents, In the elderly) in Israel (Intracerebral) (PTC Therapeutics pipeline, March 2024)
  • 29 Feb 2024 PTC Therapeutics announces intention to submit BLA to the US FDA for Aromatic amino acid decarboxylase deficiency in March 2024
Subscriber content

You need to be a logged in subscriber to view this content.

If your organization has a subscription then there are several options available to help you access AdisInsight, even while working remotely.

  • IP authentication when working within your organization’s network.
  • with username/password or try via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days.
  • Contact us at AsktheExpert.AdisInsight@springer.com for assistance.

If your organization does not have a subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Buy a PDF version of the profile.
  • Request a free trial
Back to top