FDA Approves Kalydeco for Cystic Fibrosis Patients Ages 2 and Up


The U.S. Food and Drug Administration (FDA) continues to provide new indications for Vertex’s Kalydeco (ivacaftor) for Cystic Fibrosis (CF) patients. Earlier today, it was approved for use in more than 600 patients ages 2 and older who have 1 of 5 residual function mutations that results in a splicing defect in the CF transmembrane conductance regulator (CFTR) gene.

The impetus for the approval was Phase 3 clinical data of the Vertex Pharmaceuticals drug in patients with these mutations, and it stays true to the May 2017 FDA approval of the drug for 23 other residual function mutations based on in vitro data.

CF, a genetic disorder, is often life-threatening because of the damage that can be done in patients’ lungs and digestive systems. Typical symptoms include frequent lung infections, heavy coughing and difficulty breathing. Current treatment options for the condition include management of the symptoms, and, at present, it affects an estimated 75,000 people in North America, Europe and Australia.

“In the five years since Kalydeco became the first approved medicine to treat the underlying cause of cystic fibrosis, we have been relentless in our efforts to bring this important medicine to all who may benefit,” said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex in a press release. “We will continue to pursue this goal until all people with CF have a medicine that treats their form of this serious and life-shortening disease.”

Kalydeco, the first treatment to consider the underlying cause of CF in people with CFTR gene mutations, is an oral medication intended to keep CFTR proteins at the cell surface open longer. This, in turn, will improve the carrying of salt and water across the cell membrane and assist in hydrating and clearing mucus from the airways of the patient.

Today’s approval is supported by more than 5 years of real-world clinical experience that demonstrate the drug’s established safety and efficacy profile. Vertex has also received approval for Orkami (lumacaftor and ivacaftor) in select groups of CF patients. Due to the 2 approvals and other positive data that has recently been published, the company has seen a rapid increase in its market value.

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