Source – Vertex Pharmaceuticals
On July 5, 2023, Vertex Pharmaceuticals announced that the European Commission has granted approval for the expanded use of ORKAMBI (lumacaftor/ivacaftor) in the treatment of children aged 1 to <2 years old with cystic fibrosis (CF) who have two copies of the F508del mutation in the CFTR gene. This mutation is the most common form of CF. ORKAMBI has already received regulatory approval in the United States, Great Britain, Australia, and Canada for individuals with CF and two copies of the F508del mutation, starting from the age of 1.
“This approval will offer some of the youngest children with cystic fibrosis the chance of improved outcomes, by treating their disease at a young age. With this important milestone, we move ever closer to our goal of providing medicines that treat the underlying cause of CF to all people living with the disease.”
– Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex
Following the European Commission’s approval, eligible patients in countries like Austria, Denmark, the Republic of Ireland, and Sweden, which have long-term reimbursement agreements in place, will soon have access to ORKAMBI for this expanded indication. In the UK, children aged 1 to <2 years old have had access to the treatment since the approval by the Medicines and Healthcare products Regulatory Agency (MHRA) in March 2023, thanks to a long-term reimbursement agreement. Vertex Pharmaceuticals is actively collaborating with reimbursement bodies in the European Union, Australia, and Canada to ensure access to ORKAMBI for all eligible patients.
This approval signifies a significant milestone in the landscape of cystic fibrosis treatment, particularly for young children. ORKAMBI offers hope and potential benefits for this specific age group, addressing a critical unmet medical need. It represents a step forward in improving the quality of life and outcomes for individuals with CF who carry the F508del mutation. The widespread approvals of ORKAMBI across multiple countries highlight the recognition of its therapeutic value and the growing understanding of the importance of targeting specific mutations in CF.
“CF symptoms and organ damage can manifest very early in life, so it is crucial to start treatment as early as possible. Today’s approval provides us with a medicine that gives a window of opportunity to possibly delay the onset of CF for these very young eligible children.”
– Silvia Gartner, M.D., specialist in Pediatrics and Pneumonology, Coordinator of the Pediatric Cystic Fibrosis Center, Barcelona
In the United States, the approval of ORKAMBI for individuals with CF and two copies of the F508del mutation has already made a substantial impact. It has provided a targeted treatment option for a specific subset of patients, addressing the underlying cause of the disease and helping to improve lung function and overall well-being. The approval has further solidified the position of ORKAMBI as an essential therapy in the CF treatment landscape, showcasing its efficacy and safety profile.
Overall, the approval of ORKAMBI in the European Union and its established presence in the United States reflect the positive advancements being made in the treatment of cystic fibrosis. These developments bring hope to individuals and their families, emphasizing the commitment of pharmaceutical companies and regulatory bodies to provide access to effective therapies for this challenging disease.