18 September 2020
Lactation Safety Information
Very limited published evidence of safety
Small amounts of both components in breast milk probably due to high protein binding
Long half-life of lumacaftor increases risk of accumulation in breastfed infants, although risk is negligible due to small amounts present
15 September 2019
OrkambiCystic fibrosis in homozygous F508del children aged 12 to 23 months
Licence extension / variation
Development and Regulatory status
Phase III Clinical Trials
Fixed dose combination. A corrector molecule, one of two new classes of ion channel modulators. The corrector modulators enhance the number of channels of the CFTR protein at the cell surface.
CF is the most common inherited disease in white populations. Prevalence is 1 in 2,500 newborn infants, with calculated carrier frequency of 1 in 25. Just over 10,000 people were recorded as having CF in the 2013 UK CF Registry .
Cystic fibrosis in homozygous F508del children aged 12 to 23 months