Cystic Fibrosis: Game-Changer Drug 'Kalydeco' For Young Children Will Soon Be Available

Cystic fibrosis (CF) is an inherited disease that affects mostly the organs. The persistence and ongoing infection in the lungs will eventually lead to death to a majority of patients with CF.

Ivacaftor, trade name Kalydeco, is called the game-changer drug. It is developed by Vertex Pharmaceuticals in conjunction with the Cystic Fibrosis Foundation, and is called the first drug that treats the underlying cause of Cystic Fibrosis rather than the symptoms of the disease.

Kalydeco Will Be Listed In May

The medication will be listed in May with the government in negotiations with Vertex on how to best ease the burden on families until then, Courier Mail said. Families would have to pay just about $6.30 per script as opposed to $300,000 a year to access the drug.

The Advertiser said that Kalydeco is currently listed for kids from the age of six, but more often than not, by that age, irreversible damage has already occurred, shaving years off their lives. Younger cystic fibrosis patients in Australia will get a massive reprieve this week when the government finally lists Kalydeco on the pharmaceutical benefits scheme (PBS) for 2- to 5-year-olds.

Orkambi Is Yet To Be Listed Though

Patients will go from taking 60 to 80 tablets a day in the worst circumstances, to as few as 10 tablets daily. However, at least 1,000 Australians over the age of 12 with a different mutation of cystic fibrosis will still be left waiting for a shot at having a normal life because the drug Orkambi is yet to be listed on the PBS. Orkambi is a combination drug (lumacaftor and ivacaftor) and is used to treat cystic fibrosis in adults and children who are at least six years old.

Michele Adair, CF Australia spokesperson said that Kalydeco and Orkambi can completely change the CF game. The drugs have significant health benefits, and these could also result in fewer hospitalizations which reduces the cost burden for all. Currently, CF patients who need Orkambi either have to take part in a clinical trial or pay $260,000 a year for access to the drug.

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