CHRISTMAS has come early for many Tasmanians with Cystic Fibrosis, after a subsidy for a $300,000 drug was approved last week.
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The drug, that is the closest thing to a cure for suffers, could mean people like six-year-old Lena Buonaccorsi, of Hobart, and Meg Willes, 20, of Launceston, could live normal lives.
The Pharmaceutical Benefits Advisory Committee approved the drug, called Kalydeco, on Friday, but a reasonable reimbursement price must now be determined with the US company that makes the drug and federal cabinet must then approve it to go on the PBS.
Lena is one of 200 Australians who will benefit from Kalydeco.
Her mum Megan Rozynski said the drug would take away regular hospital check- ups, the nebulizer she has to have every day, daily physio and the thought that Lena's life expectancy is only 37.
"She would have a normal life expectancy, she could work full- time and have children, whereas at the moment that is unknown," Ms Rozynski said.
"A lot of people who have CF don't work full-time and they choose not to have children because of their own health and the fact they don't know how long they have."
Kalydeco has been proven to increase life expectancy, lung function, lower sweat chloride levels and provide better digestion for sufferers with the G551D mutation.
Cystic Fibrosis Tasmania chief executive Paula Wriedt has urged the government to put the "breakthrough" treatment on the PBS as quickly as possible, as it could be the difference between life and death for many.
"Kalydeco is unique because it's the first drug that actually treats the underlying causes of CF - like reducing mucus that clogs the lungs - as opposed to just treating the symptoms," Ms Wriedt said.
"To actually have another step forward is the best Christmas gift that people living with CF could get."
While only 200 Australians have the G551D mutation, another drug in clinical trials has the potential to be used in conjunction with Kalydeco and help others like Meg Willes.
Miss Willes, who has two copies of DF50 mutation, said Kalydeco gave her hope for the future and she hoped she could keep herself well enough to benefit from it.
"It makes the hours of treatment and hundreds of pills a little easier to deal with," Miss Willes said.
Australia is one of the last developed nations to fund the US drug.
