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

Vertex Pharmaceuticals (Nasdaq: VRTX) has received approval from the European Commission for the expansion of KALYDECO® (ivacaftor) labeling. This approval allows for the treatment of infants as young as 1 month old with cystic fibrosis (CF) who possess specific mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The approved mutations include R117H, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R.

Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex, emphasized the significance of early CF treatment. Bozic stated, “Today’s approval is an important milestone for the cystic fibrosis community. Treating CF early in life can potentially slow the progression of the disease, which is why it is so important to start treatment from a very young age.”

Following regulatory approval by the European Commission, eligible patients in Austria, Czech Republic, Denmark, Ireland, Norway, Sweden, and The Netherlands will soon have access to the expanded indication of KALYDECO®. Vertex will collaborate with reimbursement authorities across the European Union to ensure access for other eligible patients. In the United Kingdom, infants aged 1 month and older have had access to this expanded indication since the end of 2023 due to MHRA approval and an existing reimbursement agreement between Vertex and the National Health Service.

Cystic fibrosis (CF) is a rare genetic disease affecting over 92,000 individuals globally. It is a progressive condition impacting multiple organs, including the lungs, liver, pancreas, and gastrointestinal tract. CF is caused by mutations in the CFTR gene, resulting in defective or missing CFTR protein, leading to various complications, primarily in the lungs.

KALYDECO® (ivacaftor) is designed to treat CF by facilitating the function of CFTR proteins at the cell surface, aiding in the transport of salt and water across cell membranes to hydrate and clear mucus from the airways. It was the first medication to address the underlying cause of cystic fibrosis in individuals with specific CFTR gene mutations.

Vertex Pharmaceuticals is a global biotechnology company focused on developing innovative medicines for serious diseases. It has approved treatments for cystic fibrosis, sickle cell disease, and transfusion-dependent beta thalassemia. The company continues to advance clinical and research programs in various diseases, including neuropathic pain, kidney diseases, diabetes, and muscular dystrophy.

Forward-looking statements in this press release reflect Vertex’s beliefs as of the date of publication. However, actual events or results may differ due to various factors. Vertex encourages readers to review the risks listed under “Risk Factors” in its filings with the Securities and Exchange Commission for a comprehensive understanding of the potential uncertainties.

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