medcelerator · clinician one-pager

First FDA-Approved Gene Therapy for AADC Deficiency

Kebilidi (eladocagene exuparvovec-tneq) — one-time intraputaminal gene therapy for aromatic L-amino acid decarboxylase deficiency

FDA Approved · United States · September 18, 2026

Condition: AADC Deficiency

Who this is for

This is for families of children (and adults) with genetically confirmed AADC deficiency from biallelic DDC changes who have achieved skull maturity and may be candidates for labelled US Kebilidi one-time intraputaminal gene therapy at a centre that specialises in stereotactic neurosurgery, coordinated through PTC Cares. It explains the first FDA-approved AADC gene therapy, the Week-48 motor-milestone results versus natural history, dyskinesia and procedure-risk honesty, how to start a centre referral conversation, and why accelerated approval and authorised-is-not-funded matter — without treating completed research as walk-in enrolment or importing a US vial. Authorized is not funded.

Next clinical step

The US Food and Drug Administration (FDA) has approved this medicine. In the United States, ask your doctor or a specialist whether it suits you. They can write a prescription. Insurance or public drug plan coverage is not guaranteed.

Ways to access this treatment

United States · Prescription
US prescription (FDA-labelled)
https://www.fda.gov/vaccines-blood-biologics/kebilidi

General resources for further research

ClinicalTrials.gov
US National Library of Medicine registry of clinical studies. Search by condition, drug, NCT number, and location. A listing is not enrolment, eligibility, or funding. Talk with the trial contact and a clinician before assuming a slot exists.
https://clinicaltrials.gov/

AI-produced discussion aid; not prescribing instructions. Approval does not establish coverage. Full article: https://medcelerator.com/articles/kebilidi-eladocagene-exuparvovec