This article is more than 5 years old. Information may no longer be current.
FDA approves Orkambi for treatment of cystic fibrosis
Click Here to Manage Email Alerts
Today, the drug Orkambi was approved by the FDA, as well as granted orphan drug designation, for the treatment of cystic fibrosis in children aged 12 years and older, according to a press release.
The drug is intended to treat patients that have two copies of the F508del mutation, which is the leading cause of cystic fibrosis. Orkambi (lumacaftor/ivacaftor, Vertex Pharmaceuticals) has only been assessed for patients with a double F508del mutation.
The FDA’s approval was based on two double-blind placebo controlled studies, which included 1,108 participants, that showed lumacaftor/ivacaftor significantly improved lung function in children aged 12 years and over when taken twice a day.
A breakthrough designation was granted for the drug after it proved to be substantially superior to current available therapies.
Common adverse effects of the drug included shortness of breath, upper respiratory tract infection, nausea, diarrhea, rash and increased menstrual abnormalities in women.
“The FDA encourages manufacturers to develop new and innovative treatments for serious rare diseases like cystic fibrosis. Today’s approval significantly broadens the availability of targeted treatments for the specific defects that cause cystic fibrosis,” John Jenkins, MD, director of the Office of New Drugs, Center for Drug Evaluation and Research, said in the release.