Pharma Focus Europe

European Commission Grants Approval for ORKAMBIĀ® (Lumacaftor/ivacaftor) in the Treatment of Cystic Fibrosis in Children Aged 1 to <2 Years

Thursday, July 06, 2023

Vertex Pharmaceuticals announced today that the European Commission has granted approval for the label extension of ORKAMBI® (lumacaftor/ivacaftor) for the treatment of children with cystic fibrosis (CF) aged 1 to under 2 years old who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is the most common form of the disease.

Dr. Carmen Bozic, the Executive Vice President of Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex, expressed excitement about the approval, stating that it provides an opportunity to improve outcomes for some of the youngest CF patients. She also mentioned that this milestone brings them closer to their goal of offering CF treatments that target the underlying cause of the disease to all individuals living with CF.

Dr. Silvia Gartner, a specialist in Pediatrics and Pneumonology and the Coordinator of the Pediatric Cystic Fibrosis Center in Barcelona, emphasized the importance of early treatment for CF, as symptoms and organ damage can appear very early in life. She believes that the approval of ORKAMBI® for this age group offers a window of opportunity to delay the onset of CF in eligible children.

ORKAMBI® has already received regulatory approval in the United States, Great Britain, Australia, and Canada for the treatment of CF patients aged 1 and above who have two copies of the F508del mutation in the CFTR gene.

Vertex has established long-term reimbursement agreements in Austria, Denmark, the Republic of Ireland, and Sweden, as well as provisions for access in the German healthcare system. This ensures that eligible patients in these countries will have access to the expanded indication for ORKAMBI® soon after receiving regulatory approval from the European Commission. Additionally, the UK has granted access to this indication for children aged 1 to under 2 years old since the Medicines and Healthcare products Regulatory Agency's approval in March 2023. Vertex will continue working with reimbursement bodies across the European Union, Australia, and Canada to ensure access for all eligible patients.

Cystic fibrosis is a rare genetic disease that affects over 88,000 people worldwide and shortens their life expectancy. It is characterized by progressive organ damage in the lungs, liver, pancreas, GI tract, sinuses, sweat glands, and reproductive tract. CF is caused by mutations in the CFTR gene, resulting in defective or absent CFTR protein. Individuals must inherit two defective CFTR genes, one from each parent, to develop CF. The most common CF-causing mutation is F508del. CFTR mutations disrupt the flow of salt and water into and out of cells in various organs. In the lungs, this leads to the accumulation of thick, sticky mucus, chronic lung infections, and progressive lung damage, ultimately resulting in premature death, often in the early 30s.

ORKAMBI® is an oral medication that combines lumacaftor and ivacaftor. Lumacaftor targets the processing and trafficking defect of the F508del-CFTR protein, increasing the presence of mature protein at the cell surface. Ivacaftor, a CFTR potentiator, enhances the transport of salt and water across cell membranes by CFTR proteins. Together, lumacaftor and ivacaftor help hydrate the airways and improve mucus clearance.

In conclusion, Vertex Pharmaceuticals' ORKAMBI® has received label extension approval in the European Union for the treatment of young children with CF who have two copies of the F508del mutation. This represents a significant step towards providing early intervention and improved outcomes for CF patients.

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