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FDA Approves Orkambi (Lumacaftor/Ivacaftor) for Cystic Fibrosis

Treatment is expected to cost $259,000 per year

The FDA has given the green light to the first drug for cystic fibrosis (CF) directed at treating the cause of the disease in people who have two copies of a specific mutation.

Orkambi (lumacaftor 200 mg/ivacaftor 125 mg, Vertex Pharmaceuticals) is indicated for the treatment of CF in patients 12 years of age and older who have the F508del mutation, which causes the production of an abnormal protein that disrupts how water and chloride are transported in the body. Having two copies of this mutation (one inherited from each parent) is the leading cause of CF.

Orkambi received FDA’s “breakthrough therapy” designation because the sponsor demonstrated through preliminary clinical evidence that the drug may offer a substantial improvement over available therapies. The FDA also reviewed Orkambi under its priority review program. A priority review is conducted within 6 months or less, instead of the standard 10 months, and is used for drugs that may offer a significant improvement in safety or effectiveness compared with available treatments in a serious disease or condition.

In addition, the FDA granted Orkambi orphan drug designation because it treats cystic fibrosis, a rare disease. Orphan drug designation provides financial incentives, like clinical trial tax credits, user fee waivers, and eligibility for market exclusivity to promote rare disease drug development.

It is estimated that Orkambi will cost $259,000 for a year’s treatment.

Speaking at a July 2 web conference, Stuart Arbuckle, chief commercial officer at Vertex Pharmaceuticals, said: “The price of Orkambi reflects a number of things. Certainly, one of them is the fundamental benefit that we see from treating the underlying cause of this serious disease. There are certainly cost offsets from the benefits Orkambi delivers. That isn’t the only input into our thinking of the benefit it delivers. We also had to take into account some of the things like the 15-year journey we’ve been on and the time and cost it’s taken us to get to this point. We took into account the eligible patient population, and of course, we want to continue to invest in new medicines for the future.”

CF affects approximately 30,000 people in the U.S. and is the most common fatal genetic disease in Caucasians. The F508del mutation is the most common cause of CF. People who have two copies of the F508del mutation account for approximately half of the CF population in the U.S.

The safety and efficacy of Orkambi were studied in two double-blind, placebo-controlled trials involving a total of 1,108 participants with CF who were 12 years of age or older with the F508del mutation. In both studies, the participants treated with Orkambi (two pills every 12 hours) demonstrated improved lung function compared with those who received placebo.

The efficacy and safety of Orkambi have not been established in patients with CF other than those with the F508del mutation. 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.

The most common adverse effects associated with Orkambi include shortness of breath, upper respiratory-tract infection, nausea, diarrhea, and rash. Women treated with Orkambi also had an increased incidence of menstrual abnormalities, such as increased bleeding.

Sources: FDA; July 2, 2015; and BioSpace; July 6, 2015.


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