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FDA approval: Kebilidi (from Catapult’s regulatory round-up for November 2024)

FDA has approvedeladocagene exuparvovec-tneq (Kebilidi) a recombinant adeno-associated virus serotype 2 (rAAV2)-based gene therapy, containing the human DDC  gene (dopa decarboxylase) indicated for the treatment of adult and paediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. 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. AADC deficiency is a rare genetic disorder that affects the production of some neurotransmitters. Affected individuals may experience symptoms such as delays in gross motor function (head control, sitting, standing, and walking), hypotonia (weak muscle tone), and developmental and cognitive delays. Kebilidi is the first FDA-approved gene therapy for treatment of AADC deficiency.

Published on the 3rd of december 2024. Read the entire Regulatory Round-up – November 2024 here.


OpenEdition vous propose de citer ce billet de la manière suivante :
adelage (12 février 2025). FDA approval: Kebilidi (from Catapult’s regulatory round-up for November 2024). ELSIBI. Consulté le 18 mars 2025 à l’adresse https://doi.org/10.58079/13axk


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