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EC Approves Vertex Pharmaceuticals' Orkambi for Youngest Cystic Fibrosis Patients

NEW YORK – Vertex Pharmaceuticals announced on Wednesday that the European Commission has approved its cystic fibrosis treatment Orkambi (lumacaftor/ivacaftor) for children who are between 1 and 2 years old.

Treatment eligible children must have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the most common cause of the rare and progressive disorder that affects the lungs, liver, and pancreas, among other organs.

In 2019, the EC approved Orkambi for the approximately 1,500 European children with cystic fibrosis between 2 and 5 years old and with two copies of the F508del mutation. The latest approval expands the availability of the drug to the nearly 300 1- and 2-year-old cystic fibrosis patients in Europe who have these mutations. Regulators in the US, Great Britain, Australia, and Canada have also approved Orkambi for F508del-mutated cystic fibrosis patients who are at least 1 year old.

"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," Carmen Bozic, chief medical officer at Vertex, said in a statement.

According to Vertex, the expanded indication of Orkambi will be available to patients in Austria, Denmark, the Republic of Ireland, and Sweden, where the firm has established long-term reimbursement agreements, as well as in Germany, where it has made provisions for healthcare access. After the UK's Medicines and Healthcare Products Regulatory Agency approved this expanded indication in March, Vertex also established a long-term reimbursement agreement in that country. The company said it will continue to work with reimbursement bodies in the EU, Australia, and Canada to ensure young children there have access to this drug.