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Drug

Eladocagene exuparvovec

Approved medicine
ResearchOngoing clinical research

Also known as AAV2-hAADC, Kebilidi.

RxNorm2698762ChEMBLCHEMBL4298189ATCA16AB26UNIIS51J6N56M7
2
Active clinical trials
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

SIngle-Stage, Open-Label, Safety and Efficacy Study of Adeno-Associated Virus Encoding Human Aromatic L-Amino Acid Decarboxylase by Magnetic Resonance MR-guided Infusion Into Midbrain in Pediatric Patients With AADC Deficiency

Clinical trial2026-08-27Results expected Q3 2027 · ClinicalTrials.gov

An Open-label Safety and Efficacy Study of AAV2-hAADC Administered by MRI-Guided Convective Infusion Using a Transfrontal Trajectory Into the Putamen and Caudate of Participants With Young Onset Parkinson's Disease

Clinical trial2025-12-05Results expected Q3 2028 · ClinicalTrials.gov

Approval: Upstaza (EMA)

Regulatory2022-07-18EMA

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

Clinical trial2022-05-16Completed · ClinicalTrials.gov

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Eladocagene exuparvovec
Aliases & brands
AAV2-hAADCKebilidi
RxNorm CUI
2698762
ChEMBL ID
CHEMBL4298189
ATC codes
A16AB26
UNII
S51J6N56M7
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2022-07-18
Latest approval
2022-07-18
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2022-07-18Approval
Approval: Upstaza (EMA)
Indication: Upstaza is indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L amino acid decarboxylase… Show full indication

Upstaza is indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L amino acid decarboxylase (AADC) deficiency with a severe phenotype (see section 5.1).

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

3 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
3
registered trials across all phases
LATEST COMPLETION 2022
2
Active studies
2
Recruiting
1
Completed
PHASE DISTRIBUTIONn = 3
Phase 12Phase 21

Recent completions

Trials that read out recently, adding to the completed evidence base.

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

CompletedPH IINational Taiwan University Hospitaln=122022
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.