EU approves expanded use of Vertex’s cystic fibrosis drug

 The European Commission has approved a label expansion for Vertex Pharmaceuticals’ (NASDAQ:VRTX) cystic fibrosis medication, KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor, broadening its use to patients aged 2 years and older with at least one non-class I mutation in the CFTR gene. This regulatory milestone makes the treatment available to approximately 4,000 additional individuals living with cystic fibrosis (CF) in the European Union.

The decision follows Vertex’s long-term commitment to addressing the needs of the CF community, including those with rare mutations. Executive Vice President Carmen Bozic expressed satisfaction with the European Commission’s decision, which she believes will allow more CF patients to benefit from the transformative effects of KAFTRIO®.

In countries such as Austria, Denmark, Ireland, Norway, and Sweden, where KAFTRIO® reimbursement agreements exist, and in Germany, where healthcare system provisions apply, eligible patients are expected to gain access to the therapy shortly. Vertex plans to continue its collaborative efforts with reimbursement authorities throughout the EU to facilitate access for all eligible patients.

Cystic fibrosis is a life-shortening genetic disease that affects over 94,000 individuals in North America, Europe, and Australia. It is a multi-organ condition that leads to progressive lung damage and other complications, with the median age of death in the 30s. However, treatments like KAFTRIO® are improving the projected survival rates.

KAFTRIO® works by increasing the quantity and function of the CFTR protein at the cell surface, which is crucial for the transport of salt and water across cell membranes. This helps to hydrate and clear mucus from the airways, addressing the fundamental cause of CF.

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