Ellie-May Jury’s family cried with joy at the news they could access a new life-changing treatment for her cystic fibrosis (CF).
The nine-year-old watched a live feed in her family’s car last Friday, as the Pharmaceutical Benefits Advisory Committee (PBAC) decided whether to list the drug.
“I was squeezing my sister’s hand asking, ‘Is it going to happen?’” she said.
The new drug, Orkambi, could add decades to the lives of 1300 Australians like Ellie-May by drastically reducing permanent lung damage, medication and hospital visits.
But at $300,000 a year in treatment costs, the drug was out of reach for most.
After years of haggling with the drug’s manufacturer, Vertex Pharmaceuticals, the committee approved the drug for the Pharmaceutical Benefits Scheme.
“I think we all just wanted to get out of the car and dance and celebrate,” Ellie-May said.
The avid surfer, singer, netballer, runner and high jumper said the drug would probably change her life.
“It will make me less out of breath and help me go further and faster. I just want to get on it straight away and see what it does.”
CF is an incurable genetic condition with an average life expectancy of 37 years.
Ellie-May will again visit hospital soon due to a nasty cold, which can be life-threatening for a person with CF.
“It’s making me cough up some disgusting mucus and the doctors just think we should get rid of it before it does some serious lung damage,” she said.
Her mum Jenifer was “over the moon” with the PBAC decision.
“Ellie-May hasn’t tried it before so we’re just excited to get her on it and see what happens,” she said.
Orkambi can help about half of people with CF, who have the condition’s most common gene mutation.
This means the drug could help half of about 40 people in Geelong who have CF.
