- The approval is based on P-III ARRIVAL study results assessing Kalydeco (ivacaftor) in 11 children with CF aged 6mos. to <12 mos. with 1/10 mutations in the CFTR gene for 24wks.
- The P-III ARRIVAL study results: mean baseline sweat chloride (101.5 mmol/L); @24wks. mean sweat chloride level (43.1 mmol/L); mean absolute change (-58.6 mmol/L); no patient discontinued therapy due to adverse events
- Kalydeco (ivacaftor, 25,50,75,150 mg) is an oral CFTR potentiator enables the longer opening of CFTR protein at the cell surface to improve the transport of salt and water across the cell membrane and is approved in the US, Canada and EU for the treatment of CF in patients aged >12mos.
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