Trikafta has been shown to be safe with potentially fewer adverse effects noted than with prior modulators. Notable drug-drug interactions include antifungal medications, certain antibiotics (such as rifampin or rifabutin), several seizure medications, and St. John’s wort. Similar to all CFTR modulators, Trikafta should be administered with fatty foods to help improve absorption. Quarterly monitoring of liver function testing in the first year of treatment and annual eye examinations are recommended to monitor for gastrointestinal (GI) complications, as well as the possibility of cataracts.
These CFTR modulators have heralded a new era of personalized medicine and life-prolonging therapies for an increased number of individuals with CF (Figure 2). Studies are now under way to assess the long-term effects of Trikafta as well as the possibility of simplifying daily treatment burden. As of the 2018 Cystic Fibrosis Foundation Patient Registry report, more than 50% of individuals living with CF are aged older than 18 years and the median life expectancy continues to rise.1
There is hope that CFTR modulators will help to continue to improve median life expectancy. Nevertheless, it is important to note that these therapies do not reverse existing disease, and approximately 10% of individuals presently do not qualify for any of the available CFTR modulator therapies based on their genotypes.1 The availability of mutation-specific modulators has brought new hope to individuals with CF and their families, but the search continues to find modulator therapies to benefit all individuals with all CFTR mutations.
Note from Dr. Lee:
The Cystic Fibrosis (CF) Foundation’s partnership with the pharmaceutical industry has significantly impacted patients with this lethal disorder by making CFTR-modifying drugs available. We look forward to the anticipated positive outcomes on morbidity and mortality for CF. Partnerships with pharma may serve as the way to accelerate the development of medicines for other disease where therapies are limited and/or less effective.
- Carlton Lee, PharmD, MPH, FASHP. FPPAG
1. Cystic Fibrosis Foundation Patient Registry. 2018 Patient Registry Annual Data Report. Bethesda, Maryland. Available at: https://www.cff.org/Research/Researcher-Resources/Patient-Registry/2018-Patient-Registry-Annual-Data-Report.pdf. Published August 2019. Accessed January 13, 2020.
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