Children's Health

First Drug to Target Cause of Cystic Fibrosis Approved

Kalydeco Approval: Personalized Medicine for CF continued...

According to Reuters, Kalydeco will cost $294,000 per year. The company says it has assistance programs in place for eligible CF patients not covered by insurance.

Only about 30,000 Americans have cystic fibrosis. But it's likely that CFTR drugs will have uses far beyond CF. For example, the drugs may be able to help the mucus buildup that's a key factor in COPD, the fourth leading cause of death in the U.S. and in Europe. CFTR may also play a role in smoke-induced lung damage.

Vertex isn't the only company working on CFTR drugs. FoldRx Pharmaceuticals, owned by Pfizer, is working on compounds that combine the CFTR corrector and CFTR potentiator functions.

A CF drug called ataluren is under development by PTC Therapeutics. Ataluren targets CFTR mutations seen in about 10% of CF patients.