Family's relief after cystic fibrosis drug gets green tick

The drug that could change Pippa's quality of life is expected to become available in coming months

EXCITING NEWS: Chris and Cassie Warwick are excited to hear that a drug called ORKAMBI has been recommended for reimbursement by PBAC. Picture: Jessica Perkins

CASSIE and Chris Warwick were blindsided just days after the birth of their daughter Pippa.

Doctors told them Pippa had cystic fibrosis.

"It was awful ... it was a shock," Mrs Warwick said.

Cystic fibrosis is a genetic disorder that predominantly affects the lungs, digestive system and reproductive system.

Last Friday, a drug called ORKAMBI was recommended for reimbursement by the Pharmaceutical Benefits Advisory Committee for those aged 2-5 and with two copies of the F508del cystic fibrosis gene mutation in Australia.

ORKAMBI is a drug trialled and tested to treat the disease to improve quality of life for many children.

Modelling suggests that with early intervention, it increases life expectancy by an average of 23 years. The drug was approved for those aged six and over last year.

Once contracts between Vertex and the Australian Government are drawn up following the recommendation, ORKAMBI will be placed on the PBS list.

It will then cost Australians $6.80-$40 depending on their income.

The drug will hopefully be available before the end of the year.

Despite Pippa being such a healthy two-year-old, Mrs Warwick said it was exciting and a relief to hear of the drug reimbursment recommendation considering they were unsure of where Pippa's health would be at in a couple of years.

"We were really holding out for that to be approved because we knew how well it was working for the older children," Mrs Warwick said.

She said it was good to know that children across both age groups would be covered.

Mrs Warwick said symptoms of the condition could vary but with Pippa it mainly caused digestive issues requiring her to have digestive enzymes when she ate.

Pippa doesn't attend daycare as the risk of contracting a disease is too high.

Mrs Warwick said she had the same concern for when her daughter was ready to start school.

"Just knowing that if she's on that drug and it is lowering her chances of getting sick and generally making her better then it's not so daunting thinking about her going to school and big public places ... because it is still something that's in the back of your head," Mrs Warwick said.

Mrs and Mr Warwick said an approval for the drug would be a step in the right direction for future medication that may help children with cystic fibrosis.

"Even if this drug isn't the best drug or it's not the wonder drug, it's definitely going to open the pathway for other drugs," she said.