Goornong family delighted as cystic fibrosis drug gets approved

A GOORNONG family said they are delighted with the news Kalydeco has been approved by the Pharmaceutical Benefits Advisory Committee for listing on the Pharmaceutical Benefit Scheme.

The medication, which is available in Australia, but at a price out of reach for most families – $300,000 a year, could all but cure six-year-old Amelia McCrohan who has a rare form of cystic fibrosis which attacks the G551D mutation.

Now, after an extensive review process, the PBAC has confirmed the profound benefits that Kalydeco offers individuals and the healthcare system are significant.

A campaign to make the drug available on the PBS has moved one step closer and is now subject to price negotiations with the sponsor company Vertex.

Amelia’s father Tim McCrohan said this is a “great achievement”.

“We have had our Christmas present come early, that is how we are looking at it,” he said.

“We have two more steps to go but our resolve has just intensified.

“Once it becomes available we will be able to lead a normal life.”

CEO of Cystic Fibrosis Australia David Jack said Kalydeco is a very expensive therapy, but the benefit it delivers to those living with the rare form of cystic fibrosis known as G551D is beyond measure.

“Our Christmas wishes have been granted, that is how the cystic fibrosis community feels after an agonizing wait for a recommendation to list Kalydeco on the PBS,” he said.”

“We are confident that once pricing negotiations conclude, the Minister for Health, the Hon Peter Dutton, will take the PBAC’s recommendation to Cabinet and deliver a listing date for the 200 Australians waiting on Kalydeco,”

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