FDA Approves First Triple Combination Therapy for Cystic Fibrosis

The FDA has given the go-ahead for Vertex Pharma’s Trikafta (elexacaftor/ivacaftor/tezacaftor) for treating patients with the most common cystic fibrosis (CF) mutation.

The agency’s decision makes the medicine the first approved by the FDA as a triple combination therapy. The approval is for patients 12 years and older with cystic fibrosis who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene — approximately 90 percent of CF patients.

The three-drug combination therapy targets the defective CFTR protein, helping protein created by the gene mutation to function more effectively.

“The incredible speed of this approval underscores our shared sense of urgency with the FDA and the CF community for bringing this medicine to eligible people with CF, particularly those without a medicine targeting the underlying cause of their disease,” a Vertex spokesperson said.