eladocagene exuparvovec-tneq
Drug Details
- Generic Name
- eladocagene exuparvovec-tneq
- Brand Names
- KEBILIDI
- Application Number
- BLA125722
- Sponsor
- PTC Therapeutics, Inc.
- NDC Codes
- 1
- Dosage Forms
- SUSPENSION
- Routes
- INTRACEREBRAL
- Active Ingredients
- ELADOCAGENE EXUPARVOVEC
Indications and Usage
1 INDICATIONS AND USAGE 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 [see Clinical Studies ( 14 )] . Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory clinical trial. KEBILIDI 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 change from baseline in gross motor milestone achievement at 48 weeks post-treatment. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory clinical trial. ( 1 , 14 )