LONDON - Vertex Pharmaceuticals (NASDAQ:VRTX) has announced the European Commission's approval to expand the use of KALYDECO® (ivacaftor) for treating cystic fibrosis (CF) in infants as young as 1 month old with specific CFTR gene mutations. This regulatory nod marks a significant development in the management of CF, a genetic disorder known to shorten life expectancy.
The approval, announced today, allows for the treatment of infants with one of nine mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. These mutations include R117H, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R. KALYDECO® is the first and only medicine approved in the EU to address the root cause of CF in this age group for these mutations.
Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex, emphasized the importance of early treatment, which can potentially slow disease progression.
Following the European Commission's approval, existing access agreements will enable eligible patients in Austria, Czech Republic, Denmark, Ireland, Norway, Sweden, and The Netherlands to access the treatment shortly. In the U.K., eligible infants have had access to the expanded indication since the end of 2023, thanks to a reimbursement agreement between Vertex and the National Health Service.
Vertex is committed to working with reimbursement authorities across the European Union to ensure all other eligible patients can access the treatment. The company continues to invest in research and development of therapies for multiple chronic genetic diseases, with a focus on cystic fibrosis, sickle cell disease, and transfusion-dependent beta thalassemia.
Cystic fibrosis affects over 92,000 individuals worldwide and is characterized by the buildup of thick mucus due to defective CFTR proteins caused by mutations in the CFTR gene. This approval is based on a press release statement from Vertex Pharmaceuticals.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.