VERTEX Pharmaceuticals has announced the TGA approval of Trikafta (elexacaftor/tezcaftor/ivacaftor and ivacaftor) for children with cystic fibrosis (CF) aged 6-11.
Eligible patients have at least one F508del mutation in the CF transmembrane conductance regulator (CFTR) gene, the most common CF-causing mutation worldwide.
The approval follows the recent PBS listing of the item for those aged 12 and older.
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