Medication for treating CF

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Vertex Begins Phase 3 Study of Kalydeco and VX-809

At the end of February 2013, Vertex Pharmaceuticals Inc. announced the initiation of international Phase 3 clinical trials of Kalydeco™ (formerly known as VX770) in combination with VX-809, a CF drug in development. Both Kalydeco and VX-809 are designed to treat the root cause of CF — a faulty gene and its protein product, CFTR. The combined therapies are designed to raise the chances of treating the underlying cause of cystic fibrosis.  

The six-month studies will examine the combination treatment in people ages 12 and older who have two copies of the Delta F508 mutation of CF, the most common mutation in cystic fibrosis. In Europe, more than 80% of the CF patients has at least one copy of the Delta F508 mutation.

The studies will be conducted at approximately 200 clinical trial sites in North America, Europe and Australia. In total, about 1,000 patients will be enrolled across the two studies, which will test two different doses.

The U.S. Food and Drug Administration (FDA) recently awarded a “Breakthrough Therapy Designation” to Kalydeco and VX-809. This new designation is intended to speed the development of select potential therapies that treat life-threatening diseases or conditions. As a result, the timeframe of the Phase 3 trial of Kalydeco and VX-809 has been considerably shortened in order to accelerate testing. Most Phase 3 trials are about one year long.

In 2012, EMA, the European Medicines Agency and the US FDA approved Kalydeco when taken alone for people with the G551D mutation of CF ages 6 and older. But in most European countries it’s not available yet because the drug is very expensive and most health authorities have not yet granted reimbursement.

People with CF and their families who have questions about the Phase 3 trial of Kalydeco and VX-809 should speak with their CF health care providers.

You can read the press release from Vertex.

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