^{Maryam Tabatabai, PharmD, is associate vice president of clinical information at Prime Therapeutics.} 

Cystic fibrosis (CF) is a life-threatening genetic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, negatively affecting the lungs and digestive system. About 40,000 children and adults in the United States live with CF. Thanks to scientific advancements, life expectancy has significantly increased well into adulthood, but there is still no cure. The health complications, impact on the quality of life and financial burden are enormous.  

Emerging treatments — including gene therapy and next-generation CFTR modulator drugs — offer new hope for improved outcomes. However, the CF drug pipeline remains dynamic, and many new therapies are still in the early stages of development and review. As with all medications, ensuring the safety and efficacy of these treatments remains a top priority. 

Here’s what you should know about the latest in CF care.  

How CF treatment has evolved 

Incremental innovation 

New CF treatments can build on therapies that already exist. For example, in December 2024, Vertex Pharmaceuticals received FDA approval for its next-generation triple-combination CFTR modulator vanzacaftor/tezacaftor/deutivacaftor (Alyftrek)¹ that builds on elexacaftor/tezacaftor/ivacaftor and ivacaftor (Trikafta), which received FDA approval in 2019.²  Alyftrek is the first fixed-dose CFTR modulator that patients only need to take once a day, making treatment adherence potentially easier. In addition to oral Alyftrek and Trikafta, prior oral CFTR modulator approvals that paved the way for more innovation include ivacaftor (Kalydeco), lumacaftor/ivacaftor (Orkambi) and tezacaftor/ivacaftor and ivacaftor (Symdeko). 

• Why this matters: Innovation offers more options to patients.  

• What health plans should know: Coverage policies should provide patients access to therapy that is clinically appropriate and consider genetic mutations. Treatment innovation isn’t cheap. New modulators may be priced at or above Trikafta’s wholesale acquisition cost of about $370,000³ a year, with similar figures for Alyftrek and Kalydeco, requiring careful budget impact modeling.  

Precision medicine 

Precision medicine involves personalizing treatment and prevention to the individual genetic makeup of a patient’s condition, as well as considering individual differences in a patient’s environment and lifestyle. Precision medicine matches the right treatment to the right patient at the right time. One-time administration gene therapies are being evaluated for CF, and may offer a new treatment modality for patients, but these investigational therapies are in early phases of study.   

• Why this matters: Targeted treatment options may provide hope especially for patients who aren’t eligible for or who don’t respond to certain CFTR modulators.  

• What health plans should know: Health plans will need to consider genetic testing infrastructure, mutation-specific coverage policies and case-by-case evaluations rooted in clinical evidence. 

Generics entering the market 

Some high-cost CF drugs may become more affordable in the coming years as they become eligible for generic versions. For example, Kalydeco from Vertex could lose its market exclusivity in 2028, opening the door for generic alternatives. Similarly, Symdeko, another CFTR modulator from Vertex, is expected to be eligible for a generic alternative by 2031. Generics for Trikafta and Alyftrek, are not expected until 2037 and 2039, respectively.  

• Why this matters: These future market changes could reduce costs for patients and health care systems, making life-changing treatments more accessible to a broader population. 

• What health plans should know: Health plans should prepare for cost savings and expanded access as high-cost cystic fibrosis drugs like Kalydeco and Symdeko approach generic availability in the next decade, while continuing to manage the financial impact of newer therapies like Trikafta and Alyftrek, which will likely remain brand-only until the late 2030s. 

Before these novel therapies are approved, health plans should be equipped with the latest drug insights. That’s why Prime Therapeutics (Prime) clinical experts share robust forecasting, trends and insights through its FDA Expected Decisions, Trending Topics & Drug Approvals, Quarterly Drug Pipeline and Quarterly Drug Approvals publications.  

As the treatment paradigm shifts, Prime will continue to monitor developments and support our clients in managing these high-cost therapies.  

For resources for CF caregivers, visit the Cystic Fibrosis Foundation 

Learn more about how Prime helps clients and members manage cystic fibrosis 

References

1. Vertex provides pipeline and business updates in advance of upcoming investor meetings. Vertex Pharmaceuticals Newsroom. (2025, January 12). https://news.vrtx.com/news-releases/news-release-details/vertex-provides-pipeline-and-business-updates-advance-upcoming-0
2. Antrim, A. (2024, December 20). FDA approves expanded use for Trikafta in cystic fibrosis. Pharmacy Times. https://www.pharmacytimes.com/view/fda-approves-expanded-use-for-trikafta-in-cystic-fibrosis
3. Nolen, S., & Robbins, R. (2023, February 7). This cystic fibrosis drug is a ‘miracle’ but these families can’t get it. https://www.nytimes.com/2023/02/07/health/cystic-fibrosis-drug-trikafta.html