By Jeff Alfonsi, Sara Henderson, Peng Hsiao and Richard B. Kim
In 2012, the U.S. Food and Drug Administration approved a new cystic fibrosis (CF) medication called Kalydeco (ivacaftor). The approval of Kalydeco was significant because it targeted one of the causal genetic mutations rather than a more traditional approach focused on symptom relief. Furthermore, the Cystic Fibrosis Foundation created a registry where patients could contribute their genetic information, which helped researchers identify the target for Kalydeco. This story represents a major shift in the development of more targeted and individualized therapeutic agents.