THE MOTHER of a 17-year-old who has a rare form of cystic fibrosis says she was devastated by a decision to defer the listing of new drug Kalydeco.
Last month the Pharmaceutical Benefits Advisory Committee (PBAC) considered the submission to list the drug on the PBS or the Life Saving Drugs Program (LSDP).
But earlier this week, PBAC told Cystic Fibrosis Australia (CFA) it was unable to make a decision until it received more information from sponsor company, Vertex.
CFA chief executive David Jack said another review may not happen until March next year.
Costing $300,000 a year, the drug is said to be the most ‘exciting new treatment’ for the rare G551D form of cystic fibrosis.
Shani Stevens, whose daughter Savannah is one of 200 Australians living with the rare form of the disease, said she felt physically ill after hearing the news.
“When I read the decision has been deferred, I found my feelings difficult to put into words – devastation does not come close,” Ms Stevens said.
Cystic fibrosis is the most common life-threatening diseases for young Australians today.
People with the disease have difficulty breathing because of the mucus that clogs their lungs and digestive system. There is no cure for the disease and life expectancy is usually about 40 years.
Cystic Fibrosis Victoria chief executive Helen McNeill earlier told The Courier Kalydeco was the most exciting new treatment for the disease.
Currently Savannah spends two hours every day, morning and night, to clear her lungs.
The teenager, who is in year 11 at Mt Clear College, takes up to 30 pills a day to manage her illness.
But, Ms Stevens said, Kalydeco had been shown to reverse the symptoms associated with cystic fybrosis.
“You just have to wait,” Ms Stevens said. “Your life is on hold on the whim of someone or something else.”