The Food and Drug Administration (FDA) has expanded the indication for Kalydeco® (ivacaftor; Vertex) to include treatment of cystic fibrosis in patients 4 months to less than 6 months of age who have at least 1 mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to ivacaftor based on clinical and/or in vitro assay data. Previously, the treatment was indicated for patients 6 months and older.
The approval was based on data from a phase 3 open-label safety study (ARRIVAL) that included a cohort of 6 children 4 months to <6 months of age with 1 of 10 mutations in the CFTR gene (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H). Findings from the study showed that the safety profile of this cohort was similar to that seen in older children and adults. Following 24 weeks of treatment, the mean absolute change from baseline in sweat chloride (a biomarker) for patients aged 4 months to <6 months (n=3) was -50mmol/L (95% CI, -93.1, -6.9).
“Initiating therapy that treats the underlying cause of cystic fibrosis as early as 4 months of age may have the potential to modify the course of the disease,” said Margaret Rosenfeld, MD, MPH, Seattle Children’s Research Institute and Department of Pediatrics, University of Washington School of Medicine.
Kalydeco, a CFTR potentiator, is available as 150mg tablets for patients 6 years of age and older, and as 25mg, 50mg and 75mg oral granules for patients aged 4 months to less than 6 years. The product is not recommended for use in children aged 4 months to less than 6 months with hepatic impairment and/or taking concomitant moderate or strong CYP3A inhibitors.
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FDA approves Kalydeco® (ivacaftor) as first and only CFTR modulator to treat eligible infants with CF as early as four months of age. https://www.businesswire.com/news/home/20200925005076/en/FDA-Approves-KALYDECO%C2%AE-ivacaftor-as-First-and-Only-CFTR-Modulator-to-Treat-Eligible-Infants-With-CF-as-Early-as-Four-Months-of-Age. Accessed September 25, 2020.