New Gene Therapy Approval Announced
The US Food and Drug Administration (FDA) has granted accelerated approval to PTC Therapeutics for its therapy, Kebilidi™ (eladocagene exuparvovec-tneq; PTC AADC), for the treatment of Aromatic l-amino acid decarboxylase deficiency (AADC).PartnerRe’s team of dedicated clinical consultants are here to help you navigate the ever-changing healthcare market and manage your risk through integrated medical management solutions that optimize clinical and financial outcomes. We work with you to limit and prohibit high-cost mark-ups on new life enhancing Gene & Cell Therapies.
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Drug Category
Gene Therapy
Drug Name
Kebilidi™ (eladocagene exuparvovec-tneq; PTC-AADC)
FDA Approval Date
11/13/2024
Anticipated Cost
Estimate of $3.8M for gene therapy plus outpatient administration.
Medical Condition
Aromatic I-amino acid decarboxylase deficiency (AADC)
FDA Approval Indication
Adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency
US Incidence &
US Prevalence
US Prevalence
AADC deficiency is an extremely rare disorder; fewer than 150 patients have been reported in the literature. Half of these cases are in Asian individuals, and a fifth are in individuals with Taiwanese ancestry. Males and females seem to be equally affected. AADC deficiency is probably underdiagnosed. The estimated prevalence in the U.S. based on cerebrospinal fluid (CSF) analysis and genetic testing is roughly 1-3:100,000 live newborns.
Key Considerations
- Outpatient
- Administration of KEBILIDI occurs through a stereotactic surgical procedure, a minimally invasive neurosurgical procedure used for the treatment of a number of pediatric and adult neurological disorders. The administration procedure is performed by a qualified neurosurgeon in centers specialized in stereotactic neurosurgery. KEBILIDI is authorized to be administered using the SmartFlow Neuro Cannula.
- Procedural complications have been reported after neurosurgery required for KEBILIDI administration. These events included respiratory and cardiac arrest which occurred within 24 hours of the neurosurgical procedure and during post-surgical care. KEBILIDI administration has the potential risk for additional procedure related adverse events including cerebrospinal fluid (CSF) leak, intracranial bleeding, neuroinflammation, acute infarction, and infection. Patients should be monitored for procedure related adverse events with KEBILIDI administration
- FDA Approval: FDA Approves First Gene Therapy for Treatment of Aromatic L-amino Acid Decarboxylase Deficiency | FDA
ICD-10 Codes
E70.81
J Codes
C9399 and J3590
For full label information and more, visit Package Insert - KEBILIDI
Our PULSE+ Plus® Clinical Consultants are here to help you navigate the process and answer your questions about all things gene and cell therapy related. Our commitment is to provide you offerings that deliver the best clinical and financial outcome, while delivering ongoing support every step of the way.
Our PULSE+ Plus® Clinical Consultants are here to help you navigate the process and answer your questions about all things gene and cell therapy related. Our commitment is to provide you offerings that deliver the best clinical and financial outcome, while delivering ongoing support every step of the way.
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