The Wall Street Journal reports that the CF Foundation played a major role in the funding of a new potential medicine -- Kalydeco (VX-770) -- and is a pioneer in driving drug development for rare diseases.
In a WSJ Health Blog, journalist Jonathan Rockoff notes that a significant challenge in developing CF treatments is identifying companies willing to invest in research for a disease that affects a small portion of the population.
The blog highlights the CF Foundation's business model, which offers millions of dollars to pharmaceutical companies to fund CF research and identify compounds to fight the disease.
"We took the risk," says Robert J. Beall, Ph.D., president and chief executive of the Cystic Fibrosis Foundation, so that companies would get involved in CF research projects and a
The drug that is furthest along in development is Kalydeco. Vertex Pharmaceuticals, the maker of Kalydeco, filed for regulatory approval of the drug on Oct. 20. If approved, it will be the first medicine on the market that targets the underlying cause of CF.