Vertex scores European cystic fibrosis medicine approval

The European Commission has granted a label expansion of the combination treatment KAFTRIO^® (ivacaftor/tezacaftor/elexacaftor) with ivacaftor, for children aged two to five years old with cystic fibrosis. The treatment from Vertex Pharmaceuticals is indicated for individuals who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CYSTIC FIBROSISTR) gene.

KAFTRIO (ivacaftor/tezacaftor/elexacaftor) with ivacaftor

The biotech company highlighted the mechanism of action for the combination regime with ivacaftor. Administered orally, it works by increasing the quantity and function of the CYSTIC FIBROSISTR protein at the cell surface.

Together, elexacaftor and tezacaftor increase the amount of the mature protein at the cell surface by binding to different sites on the CYSTIC FIBROSISTR protein. The combination of ivacaftor, tezacaftor and elexacaftor support hydration and mucus clearance from the airways.

“As cystic fibrosis starts in early childhood and is a progressive disease, it is important to treat people with cystic fibrosis as early as possible. With the approval of KAFTRIO for children as young as two years, we can now treat young children with a medicine that has the potential to slow disease progression by addressing the underlying cause of the disease,” stated Professor Dr Marcus Mall, Head of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine at Charité Universitätsmedizin Berlin.

European Commission extends approval of cystic fibrosis medicine

Expediting access for eligible patients with cystic fibrosis

According to Vertex Pharmaceuticals, due to existing reimbursement agreements in Austria, Denmark, Ireland, Norway, Latvia, and Sweden, eligible patients in these countries will be able to access to this new expanded indication just after it is approved by the European Commission.

The biotech added that for UK patients, following approval by the Medicines and Healthcare products Regulatory Agency (MHRA) on 15 November 2023, and an existing reimbursement agreement between Vertex and the NHS, children two years and older can access this expanded KAFTRIO (ivacaftor/tezacaftor/elexacaftor) indication.