Make Kalydeco Affordable for Cystic Fibrosis Sufferers in Australia

Kalydeco marks a breakthrough in the treatment of Cystic Fibrosis (CF) because it is the first medication to target the underlying cause of CF - a defective gene.
All other therapies only address the symptoms of the disease.

It is a tablet taken twice a day for the treatment of CF in people ages 6 and older with the G551D mutation. In people with this mutation, Kalydeco helps improve lung function and lower sweat chloride levels and helps patients gain weight. The G551D mutation is present in approximately 8% of the Australian CF population (about 240 people). Kalydeco has been approved for public funding in other countries where the percentage of CF sufferers with the G551D mutation is lower than in Australia, such as the United States where about 4% have the G551D mutation. Kalydeco is also available at a greatly reduced cost on health schemes in England, Scotland, Ireland and Canada.

Also, there is the potential for Kalydeco to improve the health and lives of a larger group of CF sufferers as a result of the clinical trials undertaken by the manufacturer, Vertex, on Kalydeco's effectiveness, in combination with other drugs, in treating people with the Delta F508 mutation (as well as children as young as age 2 with the G551D mutation). Results from the second part of this Phase 2 trial showed significant improvements in lung function in people with two copies of the Delta F508 mutation who received the combination treatment.

CF is the most common, genetically acquired, life-shortening chronic illness affecting young Australians today. In Australia, one in 2500 babies are born with CF, that's one every four days. There is currently no cure.

It most critically affects the lungs, pancreas and liver, however, it also affects other organs and systems such as the intestine and reproductive system. Despite advancements in the treatment of CF, only 50% of sufferers currently reach their late thirties.

Complications increase with age, requiring ever increasing levels of (self) care and support. From birth, a person with CF undergoes constant medical treatments and physiotherapy. Management and treatment of CF is lifelong, ongoing and relentless. A person with CF may consume up to 40 capsules daily to help digest food and may need to do up to three hours of airway clearance and antibiotic therapy each day. All this before they even start their normal daily activities like going to school or work.

People with CF are not encouraged to socialise with each other. The risk of cross-infection and exacerbation of lung conditions is too great. CF can be a lonely existence. They often require frequent hospitalisation and many require lung transplants to survive.

If Kalydeco is made available at a heavily subsidised, affordable cost to all CF sufferers in Australia with the G551D mutation there would be an immediate drastic reduction in the number of hospital admissions and lung transplants for these CF sufferers. Thereby easing the financial burden on the Government and taxpayers, making more donor lungs available for other people requiring transplants and reducing the number of patients on the lung transplant waiting list and waiting times.