By Justin Flynn
It was the news that Ocean Grovers Kate and Steven MacPherson had been waiting to hear since their son Brody was diagnosed with a rare form of cystic fibrosis at four weeks of age.
Brody, 3, is now eligible to receive the wonderdrug Kalydeco, which treats the cause of cystic fibrosis (CF), not the symptoms.
Previously, the drug was only available to those over five years of age and at almost $300,000 per year it was out of the MacPhersons’ reach.
But after lengthy lobbying, the Australian Government has made Kalydeco available on the Pharmaceutical Benefits Scheme for children under six, which means Brody can start life-altering treatment almost immediately.
“We’re in a state of shock and disbelief at the moment,” Brody’s mother Kate told the Ocean Grove Voice.
“We’ve been waiting three years for this. We’ve literally campaigned for this since the day we found out Brody had CF.”
Only 30 children in Australia between the ages of two and five have Brody’s form of CF.
The drug was available at a dramatically reduced cost for kids over five, which was no help to Brody.
But a recent agreement between Vertex Australia, which manufactures the drug, and the Government means that children like Brody can get immediate care.
Kalydeco is the first medicine to target the underlying cause of CF in people who have a G551D or other gating (Class III) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene 2.
“We’re ecstatic – he’s just going to have a chance at a full and happy life,” Kate said.
“We’re just so thankful they have given immediate access.
“It’s not a cure – Brody still has CF, but it’s a chance to stop the lung damage. The stress impacts on your relationship, finances and our daughter (Elisa, 6).”
Brody will be able to access the drug after he undergoes a series of tests.
“He’ll go to kinder next year in a stronger body to fight off all those bugs,” Kate said.
“You hold them back and protect them as much as you can to be able to stop the disease in its tracks. To not have to do that and just let him have a normal life is an absolute godsend.”
