Kebilidi FDA Approval History
Last updated by Judith Stewart, BPharm on Nov 14, 2024.
FDA Approved: Yes (First approved November 13, 2024)
Brand name: Kebilidi
Generic name: eladocagene exuparvovec-tneq
Dosage form: Suspension for Intraputaminal Infusion
Previous Name: Upstaza
Company: PTC Therapeutics, Inc.
Treatment for: AADC Deficiency
Kebilidi (eladocagene exuparvovec-tneq) is a gene replacement therapy for use in the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency.
- AADC deficiency is a rare genetic disorder caused by genetic mutations in the dopa decarboxylase (DDC) gene which results in the inability to synthesize dopamine. Infants with AADC deficiency experience severe physical, mental and behavioral disabilities and suffering from the first months of life, and may also experience episodes of distressing seizure-like oculogyric crises causing the eyes to roll up in the head, frequent vomiting, behavioral problems, and difficulty sleeping.
- Kebilidi is a recombinant adeno-associated virus serotype 2 (AAV2)-based gene therapy containing the human DDC gene. It is designed to correct the underlying genetic defect by delivering a functioning DDC gene directly into the putamen, increasing the AADC enzyme and restoring dopamine production.
- Kebilidi was granted accelerated approval by the U.S. Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with AADC deficiency based on the safety and clinical efficacy findings in the PTC-AADC-GT-002 ongoing global clinical trial. Confirmatory evidence will be provided from the long-term follow up of patients already treated in the study.
- Kebilidi is administered directly administered to the brain through a stereotactic surgical procedure.
- Warnings and precautions associated with Kebilidi include procedural complications and dyskinesia.
- Common adverse reactions (incidence ≥15%) include dyskinesia, pyrexia, hypotension, anemia, salivary hypersecretion, hypokalemia, hypophosphatemia, insomnia, hypomagnesemia, and procedural complications.
Development timeline for Kebilidi
Further information
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