Drug updated on 11/27/2024
| Dosage Form | Infusion (intraputaminal; 5.6×1011 vg/mL per mL [ 2.8×1011 vg/mL per 0.5 mL]) |
| Drug Class | Adeno-associated virus (AAV) vector |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of adult and pediatric patients with aromatic 13 L-amino acid decarboxylase (AADC) deficiency
Latest News
Summary
- This summary is based on the review of one randomized controlled trial. [1]
- Motor and Cognitive Function: Rapid improvements in motor and cognitive function were observed within 12 months of treatment with eladocagene exuparvovec in 26 patients without head control, sustained for over 5 years.
- Dopamine and Symptoms: Increased dopamine production was demonstrated via positron emission tomography and neurotransmitter analysis. Symptomatic improvements included mood, sweating, temperature stability, and reduced oculogyric crises.
- Age-Related Outcomes and Quality of Life: Younger age was associated with greater treatment improvements, including motor and cognitive function. Patient growth and caretaker quality of life also improved.
- No treatment-associated brain injuries were reported, and most adverse events were related to the underlying disease rather than the treatment; post-surgery complications such as cerebrospinal fluid leakage were managed with standard care.
- Mild to moderate dyskinesia occurred in most patients but resolved within a few months.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Kebilidi (eladocagene exuparvovec-tneq) Prescribing Information. | 2024 | PTC Therapeutics, Inc., NJ USA |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency. | 26Subjects F: 50% M: 50% | 2022 | Molecular Therapy |
Document Title
Sex Distribution:
F:50%
M:50%
26Subjects
Year:
2022
Source:Molecular Therapy