The company has asked the FDA for priority review of the combination therapy, which, if granted, could shorten the review timeframe from approximately 12 months to 8 months. The FDA grants priority review for several reasons, including when a potential drug is considered a major treatment advance.

The treatment is the first to combine two drugs into a single pill to address the underlying genetic cause of the disease in those with two copies of the F508del mutation. About 50 percent of people with CF in the United States have two copies of the F508del mutation and 40 percent have one copy.

“The CF Foundation is very pleased to see that this combination treatment is moving forward with possible approval in 2015,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “This is an exciting step forward in our efforts to speed development of new and effective therapies for all people with CF.”

Vertex has also submitted an application to the European Medicines Agency for approval of the drug for people with two copies of the F508del mutation in the European Union. The combination treatment has already been granted accelerated assessment in the European Union.

Approximately 8,500 people with CF ages 12 and older in the United States and 12,000 in Europe have two copies of the F508del mutation.

Results released earlier this year from late-stage clinical trials of the combination treatment in those with two copies of F508del showed that those who received the drug had significant improvement in lung function and other important health measures, including weight gain, and a reduction in the rate of pulmonary exacerbations. The combination treatment was generally well tolerated by participants who took the drug, with few adverse events reported.

Vertex plans to begin a Phase 3b clinical trial of the potential combination drug in people ages 12 and older with two copies of the F508del mutation who have severe lung disease and may benefit from the treatment prior to its potential approval. The trial will enroll a limited number of people in the first quarter of 2015.

People with CF and their families who have questions about the Phase 3b clinical trial may contact Vertex Medical Information (in the United States) at 1-877-634-8789 or medicalinfo@vrtx.com, or (outside the United States) vertexmedicalinfo@vrtx.com.

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