FDA Approves Expansion of Two CFTR Modulator Therapies to Additional Rare CF Mutations

The U.S. Food and Drug Administration (FDA) has approved the expanded use of two CFTR modulators, Trikafta (elexacaftor/tezacaftor/ivacaftor) and Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), enabling more people with CF who have rare CFTR gene mutations to access treatment.

April 1, 2026 | 2 min read

The FDA has approved Trikafta (ages 2 and older) and Alyftrek (ages 6 years and older) for people with cystic fibrosis who have any mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that produces a CFTR protein.

With this approval, approximately 800 additional people with cystic fibrosis in the U.S. are now eligible for a CFTR modulator therapy, giving them access to treatment for the underlying cause of their disease for the first time.

People with cystic fibrosis or their loved ones who want to learn whether they may now be eligible for a CFTR modulator should talk with their care team. Additional information about treatment eligibility is also available through Vertex’s Treatments Finder.

For assistance with insurance coverage or navigating costs of care related to either medication, you can get help by calling Compass at 844-COMPASS (1-844-266-7277) to speak with a case manager. You may also email compass@cff.org or submit an online service request for support.

Share this article
Topics
CFTR Modulators | Research
Recent news
130+ Advocates Urge Congress to Protect NIH Funding During 18th Annual Teen Advocacy Day
News | 2 min read
Milestones III Raises More Than $250M to Advance a Cure and Innovative CF Care
News | 3 min read
CF Foundation Commits Up to $13 Million in Additional Funding to Advance Potential Splicing Mutation Treatment
News | 2 min read