Cystic fibrosis

Kalydeco listed on PBS for children aged 12-24 months


Respiratory medicine specialists have welcome the announcement that ivacaftor (Kalydeco) will be subsidised on the PBS for children aged 12-24 months with cystic fibrosis (CF) from 1 August.

The drug is currently subsidised for about 280 patients over two years of age  who have a G551D mutation or other class III gating mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

The extension of the listing will give an additional five to ten children a year access to the treatment that would otherwise cost $300,000 a year.

Speaking at the official announcement Dr David Armstrong, Head of Paediatric Cystic Fibrosis Services at Monash Children’s Hospital, Melbourne, said it was good news for children who until recently faced a bleak future with CF and long periods of hospitalisation

“As a clinician involved in cystic fibrosis over the last 25 years I can say that the use of these medications for patients has been literally, has been literally life changing,” he said

[And] we do indeed look forward to further advances in this type of therapy as we’ve heard as the triple combinations come down the line. So a very exciting time for us all in the cystic fibrosis role,” he added.

Nettie Burke,  CEO of Cystic Fibrosis Australia said there were now drug therapies for about 50% of people with CF, but there was still a way to go with triple combination therapy sand with widening the PBS listing age criteria to allow young children to access treatment such as  lumacaftor/ivacaftor  (Orkambi)

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