Pharma Focus Europe

Vertex Receives European Commission Approval for KALYDECO® to Treat Cystic Fibrosis in Infants as Young as 1 Month Old

Saturday, April 27, 2024

Vertex Pharmaceuticals (Nasdaq: VRTX) has announced today that the European Commission has granted approval for expanding the use of KALYDECO® (ivacaftor) to include infants as young as 1 month old with cystic fibrosis (CF) who have specific mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This approval marks a significant advancement for the CF community, emphasizing the importance of early intervention in managing the disease.

Dr. Carmen Bozic, Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex, highlighted the importance of initiating treatment at a very young age to potentially slow down CF's progression.

Eligible patients in several European countries will soon have access to this expanded indication due to existing access agreements. Vertex will continue collaborating with reimbursement authorities across the European Union to ensure access for all eligible patients. In the United Kingdom, following approval by the Medicines and Healthcare products Regulatory Agency (MHRA) at the end of 2023, eligible infants aged 1 month and older already have access to this expanded indication for KALYDECO® (ivacaftor) through an existing reimbursement agreement with the National Health Service.

Cystic fibrosis (CF) is a rare genetic disease affecting over 92,000 people worldwide, characterized by progressive multi-organ complications. It is caused by mutations in the CFTR gene, resulting in defective or absent CFTR protein, leading to various symptoms affecting multiple organs, predominantly the lungs.

KALYDECO® (ivacaftor) acts as a CFTR potentiator, facilitating the function of defective CFTR proteins at the cell surface. By enhancing the transport of salt and water across cell membranes, KALYDECO® helps hydrate and clear mucus from the airways. It was the first medication designed to address the underlying cause of CF in individuals with specific CFTR gene mutations.

KALYDECO® (ivacaftor) is prescribed for individuals with CF aged at least 1 month and weighing at least 3 kg who have certain mutations in the CFTR gene. Complete product information can be found in the Summary of Product Characteristics available on



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