Babies aged 12 to 24 months will soon have PBS Kalydeco access
Very exciting news announced over the weekend!
Kalydeco, one of the few drugs that targets the underlying cause of cystic fibrosis (CF), will be made available through the Pharmaceutical Benefits Scheme (PBS) for children aged 12 to 24 months.
Kalydeco is already subsidised for people over 24 months old, but this new funding means that many young people with CF will be able to access this critically important treatment earlier.
We now wait for the outcome of the July Pharmaceutical Benefits Advisory Committee meeting, where access to another CF drug called Orkbami was on the agenda. Orkambi also targets the underlying cause for CF and is already subsidised for people aged 6 and over; but it is not currently available for children aged 2 to 5.
So, why two different drugs to treat the same underlying cause?
Each one is only effective for a subset of the CF population, dependent on their cystic fibrosis transmembrane conductance regulator (CFTR) gene mutations. With over 2,000 gene mutations identified so far, developing treatments that are effective for everyone is a challenge!
Thankfully, each new drug is providing relief to more and more people, and will hopefully accelerate the development of new treatments for all people with CF in the future.