FDA Approves Kalydeco for Cystic Fibrosis

Kalydeco approved for cystic fibrosis patients with G551D mutation

/ Author:  / Reviewed by: Joseph V. Madia, MD

(RxWiki News) The Food and Drug Administration (FDA) has approved the first cystic fibrosis drug that treats an underlying cause of the disease. This drug can lead to significant lung function improvement for some cystic fibrosis patients.

The FDA has approved Kalydeco (ivacaftor) for cystic fibrosis patients aged six and older who have a G551D mutation. Close to 1,200 cystic fibrosis patients have this G551D mutation. While Kalydeco is costly it can provide significant long-term benefits for these patients.

"Ask your doctor about cystic fibrosis mutation testing."

Cystic fibrosis is caused by a cystic fibrosis transmembrane conductance regulator (CFTR), a protein, mutation. The G551D mutation occurs within the CFTR gene. Kalydeco restores the normal function of G551D within CFTR.

Close to four percent of all cystic fibrosis patients have this G551D mutation. In clinical trials, cystic fibrosis patients treated with Kalydeco had significant lung function improvement. Kalydeco also improved weight gain, decreased hospitalization frequency and improved quality of life.

The side effects of Kalydeco were considered to mild to moderate. Side effects include the common cold, headache, upper respiratory tract infection, stomach pain and diarrhea.

In an interview that Dr. Jeff Leiden, Vertex Pharmaceuticals Inc. Chief Excutive, gave to the Wall Street Journal, Kalydeco costs $294,000 a year. The reason for the high cost is the years of research required to develop the drug, the value it provides and the number of patients it is supposed to treat. This includes cystic fibrosis treatment research and development

Health plans do cover some cost because of the improvement it provides for this group of cystic fibrosis patients. In addition to that, Vertex Pharmaceuticals Inc. will provide financial assistance, up to 30 percent, for qualified candidates who may have a high co-pay or insurance cost. Vertex will also provide case managers for patients to aid them in any financial issues.

Kalydeco's FDA approval is a major achievement according to Steven Rowe, M.D., MSPH, from the Division of Pulmonary, Allergy and Critical Care Medicine at the University of Alabama at Birmingham School of Medicine. Dr. Rowe led several studies involving Kalydeco and believes future studies may show Kalydeco can help cystic fibrosis patients who do not have the G551D mutation. 

Reviewed by: 
Review Date: 
February 2, 2012
Last Updated:
February 2, 2012