The European Cystic Fibrosis Society (ECFS) and Cystic Fibrosis Europe (CFE) have jointly submitted a request to the European Medicines Agency (EMA) to expand the approval of Kaftrio® (elexacaftor/tezacaftor/ivacaftor) for individuals with cystic fibrosis (CF) who have non-F508del CFTR variants.

You can read the full letter here: ECFS-CFE_LetterCHMP re ETI_120225

Cystic fibrosis, a genetic disorder caused by mutations in the CFTR gene, significantly impacts patients’ quality of life and life expectancy. Kaftrio®, a CFTR modulator, has shown transformative benefits for those with the F508del variant, improving lung function and overall health.

Recent data and real-world evidence suggest that Kaftrio® is also effective for many other rare CFTR variants. The ECFS and CFE emphasize the importance of considering this evidence to ensure equitable access to this life-changing treatment across Europe.

The organizations highlight the need for EMA to include data from in vitro experiments and real-world studies in their decision-making process. They advocate for the approval of Kaftrio® for all CF patients with at least one responsive non-F508del variant, potentially assessed through a short course of therapy.

The ECFS and CFE are hopeful that the EMA will positively consider this request, allowing more CF patients to benefit from Kaftrio® and improve their health outcomes.