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...





