TGA approves Vertex’s cystic fibrosis therapy 6-11 year olds

Pharma News: Vertex announced that the Australian Therapeutic Goods Administration (TGA) has approved the use of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for children with cystic fibrosis (CF) ages 6-11 years who have at least one F508del mutation in the CF transmembrane conductance regulator (CFTR) gene, the most common CF-causing mutation worldwide.

Cystic Fibrosis begins at birth and leads to cumulative health decline over time, and premature death. In paediatric patients, there is evidence of early structural lung damage, even prior to the emergence of symptoms, including those of a respiratory nature.
“This approval follows the recent PBS listing for those ages 12 years and older and demonstrates our continued commitment to broaden access to younger Australians with CF,” said Sabrina Barbic, Senior Country Manager, Australia and New Zealand, Vertex Pharmaceuticals.

“We now look forward to the outcome of the Pharmaceutical Benefits Advisory Committee (PBAC) meeting in November, at which Trikafta will be considered for reimbursement for this younger age group. We will continue to work tirelessly alongside the CF community and other stakeholders in an effort to bring Trikafta to all Australian patients who can benefit.”

Trikafta was approved by the TGA based on the results of data from a 24-week Phase 3 study, which evaluated the safety and efficacy of Trikafta in children ages 6-11 years with at least one F508del mutation.