FDA OKs First Drug to Treat Cystic Fibrosis Mutation

Vertex Pharmaceuticals’ Orkambi has been approved as the first drug intended to treat cystic fibrosis in patients who have two copies of a specific mutation, the FDA announced last week.

Orkambi (lumacaftor 200 mg/ivacaftor 125 mg) is approved for patients 12 and older who have inherited copies of the F508del mutation from both parents. The drug received breakthrough therapy designation after Vertex demonstrated that it could offer substantial improvement over available therapies. The FDA granted Orkambi orphan drug designation because of the rarity of this form of CF and reviewed it under the priority review program. 

Vertex conducted two double-blind clinical trials involving 1,108 CF patients 12 and older who have the mutation. In both studies, patients who took Orkambi demonstrated better lung function than patients who were given a placebo. The most common side effects were shortness of breath, upper respiratory tract infection, nausea, diarrhea and rash. — John Bechtel