U.S. Food and Drug Administration (FDA) has approved “Kalydeco or VX-770” (ivacaftor) by Vertex Pharmaceuticals as reported by the company on Tuesday.
As reported by FDA, Kalydeco was found effective in the treatment of cystic fibrosis, a disease which is life-shortening in nature and affects the lungs and digestive system. In the patients of cystic fibrosis, thin layer of mucus which keeps the germs away becomes thick leading to the clogged airways resulting in infections damaging the lungs. Cystic fibrosis affects almost 30,000 people in U.S. Kalydeco attacks on the defective protein causing cystic fibrosis in almost 4% of patients.
“This is a breakthrough therapy for the cystic fibrosis community because current therapies only treat the symptoms of this genetic disease,” said Dr. Janet Woodcock, head of the FDA’s drugs division.
This drug has been approved for patients of age 6 or older and the agency has approved the drug under a six-month priority review granted to medicines. It has been approved three months ahead of the agency’s target deadline of April 18.
Kalydeco is the second approved drug by Vertex Pharmaceuticals, which got approval in U.S. in less than nine months. First was Incivek for hepatitis C treatment.
The drug has been granted an Orphan drug status by FDA as the disease is so rare.
The company has reported that Kalydeco will be shipped to U.S. pharmacies this week.