Kalydeco was recently approved to treat additional cases of gene mutations in cystic fibrosis patients, the U.S. Food and Drug Administration reported.
Previously, Kalydeco only treated 10 mutations, but since the update it can now treat up to 33.
“Many rare cystic fibrosis mutations have such small patient populations that clinical trial studies are not feasible,” Dr. Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, said. “This challenge led us to using an alternative approach based on precision medicine, which made it possible to identify certain gene mutations that are likely to respond to Kalydeco."
Patients who suffer from cystic fibrosis are affected in a variety of ways including their mucus and sweat glands and digestive tracks. In a non-affected person who does not have cystic fibrosis, fluids easily travel throughout the body with the help of ions. However, for those suffering, these fluids cannot be regulated. Because of that, patients could develop “severe respiratory and digestive problems,” along with infections.
Kalydeco is available for patients in a pill or granules, and they are instructed to take them twice a day with food.
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