Vertex Announces Medsafe Consent For TRIKAFTA®To Treat Children With Cystic Fibrosis Ages 2-5 Years

Vertex Pharmaceuticals today announced that the New Zealand Medicines and Medical Devices Safety Authority (Medsafe) has granted consent for the expanded use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of cystic fibrosis to include children aged 2-5 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, and those aged 2 years and older with a mutation in the CFTR gene that is responsive to treatment based on clinical and/or in vitro data.

Clinical manifestations of CF can appear from infancy, with evidence of structural lung damage, pulmonary inflammation and infection in paediatric patients, even before the emergence of symptoms.ii,iii To change the trajectory of CF disease progression and prevent cumulative health decline over time and premature death, early treatment intervention is critical.ii,iii

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“We welcome Medsafe’s consent to expand the indication of TRIKAFTA to include children as young as two, as well as those with a responsive mutation. It marks further progress towards our mission of providing a treatment for all people living with CF regardless of age or genotype,” said Kasia Siwek, Medical Director, Australia and New Zealand, Vertex Pharmaceuticals.

Notes:

TRIKAFTA was first consented by Medsafe in 2021 for use in people with CF aged 6 years and older with at least one F508del mutation in the CFTR gene.

From today, the updated indication for TRIKAFTA is as follows: TRIKAFTA is indicated for the treatment of cystic fibrosis in patients aged 2 years and older who have at least one F508del mutation in the CFTR gene or a mutation in the CFTR gene that is responsive based on clinical and/or in vitro data.

TRIKAFTA is currently funded by Pharmac under special authority criteria for the treatment of cystic fibrosis in patients aged 6 years and older who have at least one F508del mutation in the CFTR gene, or a mutation in the CFTR gene that is responsive to treatment based on clinical and/or in vitro data. It is not currently funded for children aged 2-5 years.

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