Trikafta Update

 

Cure4CF will not rest until there is a cure for everyone with CF.

We are thrilled the Pharmaceutical Benefits Advisory Committee has recommended the inclusion of Trikafta on the Pharmaceutical Benefits Scheme (PBS) for children aged 6-11 with one F508del mutation of the CFTR gene.

We hope that the Department of Health and the drug manufacturer move quickly, so that the modulator therapy can be accessed rapidly and affordably on the PBS by 90% of people with CF in Australia over the age of 6.

We hope this will mean by April 2023, Trikafta will hit the clinic for children aged 6-11.

Modulator therapies like Trikafta work by restoring or increasing the CFTR protein levels in people with certain mutations.  While we don’t know the full impact of modulator therapy, early results show great improvements in lung function and reduced exacerbations in many people. By improving CFTR levels, modulator therapies impact hydration of the mucus lining the airway which is thought to also improve bacterial clearance.  This means that people taking a modulator therapy may find they experience fewer new bacterial infections because of this improvement in their ability to sweep the bacteria out of the airway.

However, the impact of modulators on both the bacteria already living in the lungs and the lung damage they have caused is not yet known.  Data suggests children as young as 3 years old with CF can have bronchiectasis which is irreversible damage and scarring of the airways. For those who have experienced CF for a long time before being able to restore CFTR function, there is likely to be the need for additional therapies to attain good health.

Since 2010, Cure4CF have funded over $3.3M towards projects focused on preventing and fighting lung disease.

Our hope is that the youngest Australians now eligible for Trikafta will not develop chronic bacterial infections and lung scarring and will live longer and healthier lives.

Today’s announcement warrants celebration as a momentous step towards better health for many young Australians!

Cure4CF and our team remain all for the fight and will not give up until everyone receives the effective treatments they deserve., including children under 6 and the more than 350 people in our CF community who don’t have a F508del mutation or don’t respond well to Trikafta.

Research is still needed to find a cure that can be applied at or even before birth that is both easy to access and is lifelong. As Australia’s largest private funder of cystic fibrosis research, we will continue to work on behalf of the entire CF population.

Cure4CF will not rest until there is a cure for everyone with CF.

We are thrilled the Pharmaceutical Benefits Advisory Committee has recommended the inclusion of Trikafta on the Pharmaceutical Benefits Scheme (PBS) for children aged 6-11 with one F508del mutation of the CFTR gene.

We hope that the Department of Health and the drug manufacturer move quickly, so that the modulator therapy can be accessed rapidly and affordably on the PBS by 90% of people with CF in Australia over the age of 6.

We hope this will mean by April 2023, Trikafta will hit the clinic for children aged 6-11.

Modulator therapies like Trikafta work by restoring or increasing the CFTR protein levels in people with certain mutations.  While we don’t know the full impact of modulator therapy, early results show great improvements in lung function and reduced exacerbations in many people. By improving CFTR levels, modulator therapies impact hydration of the mucus lining the airway which is thought to also improve bacterial clearance.  This means that people taking a modulator therapy may find they experience fewer new bacterial infections because of this improvement in their ability to sweep the bacteria out of the airway.

However, the impact of modulators on both the bacteria already living in the lungs and the lung damage they have caused is not yet known.  Data suggests children as young as 3 years old with CF can have bronchiectasis which is irreversible damage and scarring of the airways. For those who have experienced CF for a long time before being able to restore CFTR function, there is likely to be the need for additional therapies to attain good health.

Since 2010, Cure4CF have funded over $3.3M towards projects focused on preventing and fighting lung disease.

Our hope is that the youngest Australians now eligible for Trikafta will not develop chronic bacterial infections and lung scarring and will live longer and healthier lives.

Today’s announcement warrants celebration as a momentous step towards better health for many young Australians!

Cure4CF and our team remain all for the fight and will not give up until everyone receives the effective treatments they deserve., including children under 6 and the more than 350 people in our CF community who don’t have a F508del mutation or don’t respond well to Trikafta.

Research is still needed to find a cure that can be applied at or even before birth that is both easy to access and is lifelong. As Australia’s largest private funder of cystic fibrosis research, we will continue to work on behalf of the entire CF population.