Ellie Haikalis is counting down the days until December 1.
On that day, the Hoppers Crossing resident will be able to access a life-saving drug for a rare mutation of cystic fibrosis.
Ellie, 12, is so happy to finally be able to get Kalydeco that she plans to postpone her birthday celebrations on November 29 until she has taken the drug and is feeling better.
The winning battle for Kalydeco to be placed on the Pharmaceutical Benefits Scheme and receive a federal government subsidy has taken more than two years.
The drug helps cystic fibrosis sufferers with a rare gene, G551D, that does not allow salt and fluid to flow through their bodies.
The result is a build-up of sticky mucus in the lungs and digestive system.
About eight per cent of cystic fibrosis patients in Australia – about 200 people – have the gene.
Kalydeco addresses the underlying causes of G551D, allowing patients to have a better quality of life. It costs $300,000 a year.
At the age of 11, Ellie was diagnosed with cystic fibrosis in August last year .
Without Kalydeco, she has been taking 20 tablets and three inhalers a day in a bid to stay healthy.
Her mum, Rachelle, said the family was relieved that the drug had finally been added to the PBS.
“It will help Ellie so much. It’s the best thing that could have happened,” she said.
“When Ellie was first diagnosed, we were told we would probably wait two to four weeks for Kalydeco to be added to the PBS. That was last August.”
Mrs Haikalis says she’s proud of how Ellie has handled the long wait. “She is such a strong girl.”
