Experts believe that of the 30,000 people who are affected in the United States by Cystic Fibrosis, an estimated 1,200 of them possess the G551D mutation. FDA Commissioner Margaret A. Hamburg, M.D comments:
Kalydeco is an excellent example of the promise of personalized medicine - targeted drugs that treat patients with a specific genetic makeup. The unique and mutually beneficial partnership that led to the approval of Kalydeco serves as a great model for what companies and patient groups can achieve if they collaborate on drug development."
Kalydeco was approved earlier than expected when reviewed by the FDA, after two 48-week placebo-controlled clinical studies had taken place. In the study, 213 patients were analyzed. One of the studies was conducted on patients 12 years of age and older, and the other on patients 6 to 11 years old. Both of the studies revealed the safety and effectiveness of Kalydeco. It had a great impact on improved lung function. The pill, when taken twice daily accompanying a food containing fat, is used to aid the protein made by the CFTR gene function more effectively, and therefore improves many of the signs and symptoms of CF, including rapid weight gain and lung function.
Jane Woodcock, M.D., director of the FDA's Center for Drug Evaluation and Research said:
Kalydeco is the first available treatment that targets the defective CFTR protein, which is the underlying cause of cystic fibrosis. This is a breakthrough therapy for the cystic fibrosis community because current therapies only treat the symptoms of this genetic disease.
The researchers explain that Kalydeco only works in patients with the G551D mutation, and does not work in the most common form of CF, which occurs in patients who have two copies of the F508 mutation in the CFTR gene. They say that an FDA approved test should be taken before patients with CF take Kalydeco, if their mutation status is unknown.
Kalydeco is manufactured by Vertex Pharmaceuticals Inc. of Cambridge Massachusetts and side effects of Kalydeco may occur. The most common are stomach aches, headaches, respiratory tract infection, diarrhea, rash, and dizziness.