Pipeline Update: Cystic Fibrosis

Oct 7, 2014
New drugs to treat cystic fibrosis are estimated to significantly influence drug spend by the end of 2015.
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  • Cystic Fibrosis

Cystic fibrosis, an orphan condition, is caused by a gene mutation and affects about 30,000 people in the U.S. It is the most common lethal genetic condition in the Caucasian population – 1 in 31 people are carriers.

The gene mutation leads to a poorly functioning ion channel, the cystic fibrosis transmembrane conductance regulator (CFTR), causing the body to produce thick, sticky mucus that builds up in the lungs and causes respiratory problems and infections. It also interferes with pancreatic emptying, which disrupts how foods are digested and absorbed.

The predicted median age of survival for someone with cystic fibrosis is in the early 40s, and pulmonary dysfunction accounts for about 85% of the deaths from this disease.

Rapidly Evolving Treatments

Previous treatment options for cystic fibrosis included inhaled antibiotics and Pulmozyme® (dornase alfa), an inhaled medication that treats the symptoms of cystic fibrosis by thinning the mucus in the lungs. In 2002, the Food and Drug Administration approved Kalydeco® (ivacaftor), the first drug to treat the underlying cause of cystic fibrosis. However, it is only approved to treat certain gene mutations accounting for about 5% of the cystic fibrosis patient population.

Other novel therapies are in development, including lumacaftor which is a mucus clearance enhancer and CFTR corrector. When combined with Kalydeco, it is expected to treat the most common mutation of cystic fibrosis and could benefit up to 50% of all patients.

While the new drugs represent an advance in care, they are likely to come at a high price tag. The cost of Kalydeco alone is more than $300,000 per patient per year. The cost of the Kalydeco/lumacaftor combination treatment is not yet known, but it is expected to dramatically increase spending on treatment for this condition.

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