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The drug was reviewed under the FDA’s priority review program, which evaluates a drug “that may offer significant improvement in safety or effectiveness in treatment over available therapy in a serious disease or condition” in 6 months, instead of the usual 10 months, the FDA statement said. Orkambi has also been designated as an orphan drug, because it is used to treat a rare disease.
The wholesale acquisition cost of Orkambi is $259,000 per year. Vertex will offer a co-pay assistance program for patients with commercial insurance, and a free medicine program for uninsured patients who qualify, the company announced during a telephone briefing after the approval announcement. There are an estimated 8,500 patients aged 12 and older with CF with 2 copies of the F508del mutation in the United States; about 35%-40% are on Medicaid, and the majority of the remainder have commercial insurance, according to the company.
Dr. Giusti had no disclosures. Dr. Millard, an investigator in past and current trials of Orkambi, said she had no other disclosures.
emechcatie@frontlinemedcom.com
This article was updated on 7/6/2015.