Quick answers
- What is Eladocagene exuparvovec-tneq used for?
- The FDA-approved label (Kebilidi, PTC Therapeutics, Inc., June 11, 2026) says: “KEBILIDI (eladocagene exuparvovec-tneq) is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. This indication is approved under accelerated approval based on the change from baseline in gross motor milestone achievement at 48 weeks post treatment […]. …” This page is not medical advice. Talk to your doctor or pharmacist before you start, stop or change any medicine.
- Does Eladocagene exuparvovec-tneq have a boxed warning?
- No. None of the 1 FDA labels we found for this medicine has a boxed warning.
- Is there a shortage of Eladocagene exuparvovec-tneq?
- No current shortage is listed by the FDA for the products of this medicine (FDA data from October 30, 2026).
- Has Eladocagene exuparvovec-tneq been recalled?
- No recall linked to the products of this medicine appears in the FDA enforcement reports we use.
- Who makes Eladocagene exuparvovec-tneq?
- 1 company lists prescription products of Eladocagene exuparvovec-tneq in the FDA NDC Directory, including PTC Therapeutics, Inc..
What Eladocagene exuparvovec-tneq is used for
Quoted from the “Indications and Usage” section of the FDA-approved label.
KEBILIDI (eladocagene exuparvovec-tneq) is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. This indication is approved under accelerated approval based on the change from baseline in gross motor milestone achievement at 48 weeks post treatment […]. …
— FDA label: Kebilidi, PTC Therapeutics, Inc., effective June 11, 2026. Read the full label on DailyMed →
This page is not medical advice. Talk to your doctor or pharmacist before you start, stop or change any medicine.
Boxed warning
A boxed warning (“black box”) is the FDA’s strongest warning on a drug label, for serious or life-threatening risks.
The label quoted above has no boxed warning.
Forms and how it is given
| Dosage forms | Suspension |
|---|---|
| Route | Intracerebral |
| Prescription products | 1 in the FDA NDC Directory |
| Brand names | Kebilidi |
Companies that list this medicine
Labelers in the FDA NDC Directory: the company named on the package (maker or distributor), with the number of prescription products.
- PTC Therapeutics, Inc.1 product
FDA approval history
- BLA: Biologics License Application: how a biologic medicine (made from living cells) is approved.
| Application | Sponsor | Approved | Later changes approved |
|---|---|---|---|
| BLA125722 | — | Date not on file | — |
Shortage status
The FDA drug shortage list does not show these products.
Recalls
No recall linked to these products (by their NDC code) appears in the FDA enforcement reports.
Official identifiers
| RxCUI (RxNorm) | 2698771, 2698778 |
|---|---|
| SPL set ID of the quoted label | 9d6a6401-c6b5-4f29-af11-67707d249482 |
| FDA labels (SPL) | 1 |
| FDA applications | BLA125722 |
| Active ingredients (FDA) | ELADOCAGENE EXUPARVOVEC |
Sources
- FDA-approved label on DailyMed (National Library of Medicine) — Kebilidi, PTC Therapeutics, Inc., effective June 11, 2026; via openFDA, FDA data exported October 30, 2026, retrieved October 31, 2026
- NDC Directory (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
- Drugs@FDA (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
- Drug recall enforcement reports (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 27, 2026, retrieved October 31, 2026
- Drug shortages (openFDA) — U.S. Food and Drug Administration (FDA), FDA data exported October 30, 2026, retrieved October 31, 2026
openFDA data is provided by the FDA as is; it may be incomplete. Always rely on the label that comes with your medicine. FDA Enforcement Report search →