Kalydeco, the first drug that targets the defective protein behind a rare form of the deadly lung disorder cystic fibrosis, was approved Tuesday by the U.S. Food and Drug Administration.
One patient advocate group applauded the decision.
“Today marks an important milestone in our journey to find a cure for cystic fibrosis,” Robert Beall, president and CEO of the Cystic Fibrosis Foundation, said in a news release. “Kalydeco addresses the underlying cause of cystic fibrosis, and the science behind the drug has opened exciting new doors to research and development that may eventually lead to additional therapies that will benefit more people.”