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# Vertex Cystic Fibrosis Drug Approved
- URL: https://www.fdaweb.com/vertex-cystic-fibrosis-drug-approved/
- Published: 2019-10-21T12:00:00.000Z
- Updated: 2026-09-15T01:57:59.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5145298

FDA has approved a Vertex Pharmaceuticals NDA for Trikafta (elexacaftor/ivacaftor/tezacaftor), the first triple combination therapy to treat patients with the most common cystic fibrosis mutation. Trikafta gained approval 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, which is about 90% of the cystic fibrosis population.

Approval was based on data from two trials. “The first trial was a 24-week, randomized, double-blind, placebo-controlled trial in 403 patients who had an F508del mutation and a mutation on the second allele that results in either no CFTR protein or a CFTR protein that is not responsive to ivacaftor or tezacaftor/ivacaftor alone,” an [agency release](https://www.fda.gov/news-events/press-announcements/fda-approves-new-breakthrough-therapy-cystic-fibrosis?ref=fdaweb.com) says. “The second trial was a four-week, randomized, double-blind, active-controlled trial in 107 patients who had two identical F508del mutations.” The safety profile of Trikafta is based on data from the 510 patients in the two trials. “The safety profile was generally similar across all subgroups of patients,” it adds.