Eladocagene exuparvovec
Orphan Drug Cold Chain RequiredEMA Approved (2022)
Contents
Description
Eladocagene exuparvovec is a gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency, an ultra-rare genetic disorder affecting dopamine synthesis. It delivers the DDC gene directly to the brain via stereotactic surgery. This one-time treatment has shown sustained motor improvements in pediatric patients with severe AADC deficiency.
Indications & Therapeutic Use
Aromatic L-amino acid decarboxylase (AADC) deficiency
Global Availability (4 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Eladocagene exuparvovec
| Generic Name | eladocagene exuparvovec |
| Brands | 1 brand available |
| Active Ingredient | eladocagene exuparvovec |
| Drug Class | Aromatic L-amino acid decarboxylase (AADC) deficiency |
| Manufacturer | PTC Therapeutics |
| Dosage Forms | Intraputaminal infusion, 1.8×10^11 vector genomes |
| Medical Code | N07XX |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Lead Time | 45 days |
| Reg. Status | EMA Approved (2022) |
| Clinical Trial | NCT02926066 |
| Countries | 4 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations5 Validated Nodes