Research & Development
EU Extends Marketing Authorization for Orkambi to Children with Cystic Fibrosis
12 January 2018 - - Boston, Massachusetts-based serious disease therapeutics developer Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) has received from the European Commission an extension of the marketing authorization for Orkambi(lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis in people with two copies of the F508del mutation, to include children ages 6 through 11, the company said.
In Europe, there are approximately 3,400 children ages 6 through 11 with two copies of this mutation.
Existing reimbursement agreements in countries such as Ireland will enable rapid access to Orkambi for these children. In other countries across the European Union, Vertex will now begin the country-by-country reimbursement process.
The European Commission's decision is based on data from two Phase 3 studies of Orkambi in children with CF ages 6 through 11 who have two copies of the F508del mutation.
One of these studies demonstrated statistically significant improvements in lung function among children treated with Orkambi compared to placebo.
In people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface. Patients with two copies of the F508del mutation are easily identified by a simple genetic test.
Orkambi, a twice daily oral tablet, is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface.
Vertex is a global biotechnology company focused on transformative medicines for people with serious and life-threatening diseases.
In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.The company has research and development sites and commercial offices in the US, Europe, Canada, and Australia.