CFE is pleased to let you know that European Commission (EC) has today (23 November) granted approval for the label expansion of Kaftrio for the treatment of children with cystic fibrosis (CF) aged 2 to 5 years old who have at least one F508del mutation in the CFTR gene. This is good news for European countries whose patients already have access to Kaftrio (previously restricted to children 6 years and over). Actual access on this label extension in individual countries will of course depend on the national/regional drug access and reimbursement procedures in place.
For example in countries such as Austria, Denmark, Ireland, UK, Norway, Latvia, and Sweden, eligible patients aged 2-5 will have access to Kaftrio shortly because they have some form of pipeline reimbursement agreement in place, which ensures earlier access to extensions  of drug therapies.
This welcome decision by the EC follows on from the approval of the CHMP committee of the EMA in July 2023.
CFE will continue to fight for those European countries that have no or limited access to CFTR Modulators such as Kaftrio and for those with rarer mutations, some of whom who could benefit from Kaftrio, but are not Included in the present EMA license. Its also important to note that because of genotype, some people with CF will never be able to benefit from a modulator. CFE will continue to urge research into further drug therapies.