Seth Piefke’s Christmas wish has come true with a $300,000 drug now within reach to save his little brother’s life.
Christmas celebrations have started early for the Piefke family of Thornton – and the cystic fibrosis community – with the Pharmaceutical Benefits Advisory Committee recommending a government subsidy for the breakthrough medication Kalydeco.
The drug will ultimately save the life of 200 Australians – including Flynn Piefke – each living with the rare CF gene mutation known as G551D.
“This has been our only wish this Christmas and it has come true,” Flynn’s mother, Leia Piefke, said.
“We are so very happy. This is a massive step forward and it’s all we could have wished for. We have had to fight very hard for this and now it’s finally within reach and there are so many beautiful little ones that will benefit from this.”
Just last week the family joined a national campaign calling for Kalydeco to be included in the Pharmaceutical Benefits Scheme.
As part of the push, Seth, 7, wrote a heart-wrenching letter to Santa telling him of his wish for Flynn, aged two-and-a-half years.
“Kalydeco is a very expensive therapy, but the benefit it delivers to those living with the rare form of CF is beyond measure,” Cystic Fibrosis Australia CEO David Jack said.
Kalydeco has been described as the most important development in the treatment of CF since the discovery of the CF gene in 1989.
Following an extensive review process, the PBAC has confirmed that, at the right price, the profound benefits that Kalydeco offers individuals, our community and the healthcare system are significant.
“Our Christmas wishes have been granted, that is how the cystic fibrosis community feels after an agonising wait for a recommendation to list Kalydeco on the PBS,” Mr Jack said.
“We are confident that once pricing negotiations conclude, the Minister for Health will take the PBAC’s recommendation to cabinet and deliver a listing date for the 200 Australians waiting on Kalydeco.”
Kalydeco is the first medicine to target the genetic defect that causes CF and can radically improve the lives of those affected.
It also paves the way for future drugs that might improve the life expectancy of people living with CF.
CF threatens the lives of more than 3000 Australians. People with the illness have difficulty breathing because of mucus that clogs their lungs and digestive systems.
“We now wait and see if the new year comes with an early PBS listing date,” Mr Jack said.