FDA approves cystic fibrosis drug Orkambi

SILVER SPRING, Md. — The Food and Drug Administration recently approved Vertex Pharmaceuticals’ Orkambi (lumacaftor 200 mg/ivacaftor), a new cystic fibrosis treatment that received designation from the FDA as a breakthrough therapy and as an orphan drug.

Orkambi is specifically for individuals with cystic fibrosis with a mutation (F508del) that disrupts water and chloride transportation in the body by producing an abnormal protein. The leading cause of the disease is when an individual inherits one copy of this mutation from each parent.

“The FDA encourages manufacturers to develop new and innovative treatments for serious rare diseases like cystic fibrosis,” John Jenkins, director of the Office of New Drugs at the Center for Drug Evaluation and Research said. “Today’s approval significantly broadens the availability of targeted treatments for the specific defects that cause cystic fibrosis.”

The FDA approved Orkambi after a priority review, which is conducted in a shorter time than most reviews for drugs the agency believes can offer significant safety and efficacy improvements over currently available drugs.