FDA Clears Combo Drug Orkambi for Cystic Fibrosis

Megan Brooks


July 02, 2015

The US Food and Drug Administration (FDA) has approved the first drug for cystic fibrosis (CF) directed at cause of the disease in individuals with two copies of the F508del mutation, the agency said today.

The drug, Orkambi (Vertex Pharmaceuticals), combines the approved drug ivacaftor (Kalydeco, Vertex) with lumacaftor and is indicated for patients with CF aged 12 years and older.

CF affects about 30,000 people in the United States. The F508del mutation is the most common cause of CF, and people who have two copies of this mutation (one inherited from each parent) account for roughly half of the CF population in the United States.

Orkambi, an orphan drug, received breakthrough therapy designation based on preliminary evidence that it may offer a substantial improvement over available therapies. The drug was reviewed under the priority review program.

In two double-blind, placebo-controlled clinical trials involving 1108 people with CF aged 12 years and older with the F508del mutation, treatment with Orkambi (two tablets every 12 hours) led to improved lung function compared with placebo.

The most common adverse effects with Orkambi include shortness of breath, upper respiratory tract infection, nausea, diarrhea, and rash, and in women, increased menstrual abnormalities such as increased bleeding.

"The efficacy and safety of Orkambi have not been established in patients with CF other than those with the F508del mutation," the FDA noted in a news release. "If a patient's genotype is unknown, an FDA cleared CF mutation test should be used to detect the presence of the F508del mutation on both alleles of the CFTR gene," they advise.

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

The Boston Globe, located in Vertex's hometown, reported that the company plans to charge about $259,000 per patient annually and plans to start shipping the drug to US care centers within days.

An FDA advisory committee recommended approval of Orkambi back in May.


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