Cystic fibrosis sufferers win affordable drug lifeline


Madeline Toki at 6-days-old suffers from cystic fibrosis. Picture credit: Instagram – marytoki84

A new cystic fibrosis drug has been listed onto the Pharmaceutical Benefits Scheme, saving sufferers hundreds of thousands of dollars a year and providing a lifeline for many.

After months lingering on waiting lists, a triple combination drug, called Symdeko, was approved last month and will be available to patients with a script from Dec 1 this year.

Cystic Fibrosis is a disease which affects both the respiratory system and the digestive system and causes thick, sticky mucus to block passageways in the lungs.

Sufferers of CF spend their lives in and out of hospital, taking drugs to allow them to clear their airways and breathe freely.

Symdeko works by targeting defective proteins that either don’t open properly or aren’t sitting correctly on the surface of a cell – causing an imbalance of salt and water and causing problems with the lungs.

The drug is safe for children over the age of six.

Cystic Fibrosis Western Australia’s registered nurse Sharon Dewar says that the researchers found that Symdeko was a better drug compared to the other medicines on the PBS.

“It has a larger reach in terms of which mutations it is compatible with, so more people are able to get the help that they need” Mrs Dewar says.

Australians will have access to three cystic fibrosis drugs by next month and Federal Health Minister Greg Hunt announced it will be made available to the public at $40.30 per script with concessional patients paying just $6.50.

Without government funding, the drug costs $273,938.

While Symdeko may be a miracle drug for some sufferers, for others like reality star Luke Toki daughter Madeline – there is still no drug that will combat the disease.

Luke, a two-time Survivor contestant, and his wife Mary say they shocked to discover during their third pregnancy that their baby girl would most likely be born with the life-threatening disorder.

“After heaps of invasive tests, the doctors found that Madeline had both a common and a rare gene mutation,” Mr Toki said.

Because of her rare gene mutation, there are no drugs in Australia which can help combat or relieve her symptoms of CF.

On October 21, the United States had a new drug Trikafta approved by the Food and Drug Association, which is expected to be a therapeutic option for 90 per cent of CF sufferers.

It us unknown if or when Trikafta will make its way to Australia, however the Toki’s are hopeful that this drug will be the cure that little Madeline desperately needs to have a healthy future.