CFI are delighted to share the following announcement from Vertex Pharmaceuticals relating to the expansion of Kaftrio to people with CF with rare mutations:

“Vertex today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label expansion of KAFTRIO^® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor for the treatment of people with cystic fibrosis (CF) aged 2 years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.”

The CHMP met earlier this week and voted to adopt a positive opinion on the label expansion. This is the first step toward potential EMA approval for those with rare mutations across Europe.

CFI CEO, Sarah Tecklenborg, “This is an incredible step forward in the pursuit of access to modulators for those with rare mutations who may benefit from them but who don’t currently have access. While it is an extremely positive development, there is still a significant process to follow.

“CFI, CF Europe, the Access to Medications Working Group, CF Clinical teams and the CF Community have campaigned for broader access to these lifesaving therapies. We will continue to come together to campaign and advocate with our members until everyone who stands to benefit from modulator therapy has access.”

To read the full press release from Vertex visit: Vertex Receives CHMP Positive Opinion for Expanded Label for KAFTRIO® in Combination With Ivacaftor for People With Cystic Fibrosis to Include Rare Mutations | Vertex Pharmaceuticals Newsroom