CAMBRIDGE, Mass. — Vertex Pharmaceuticals has filed for Food and Drug Administration approval of a drug for cystic fibrosis, the company said.
Vertex announced the submission to the FDA of an application for approval and priority review of Kalydeco (ivacaftor), which it said was potentially the first medicine to target the underlying cause of cystic fibrosis, namely mutations in the CFTR gene that causes defective or missing CFTR proteins that inhibit the flow of salt and water across cell membranes and lead to mucus buildup.
