Therapy Areas: Hereditary Disorders
French Authorities Approve National Reimbursement of Orkambi for Eligible People Ages Two and Older with Cystic Fibrosis
21 November 2019 - - The French Authorities (Comité économique des produits de santé, or CEPS) have approved national reimbursement of Orkambi (lumacaftor/ivacaftor) for people ages two and older with cystic fibrosis who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene, US-based biotechnology company Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) said.
Orkambi will be available for all eligible patients once the agreement has been published in the French Official Journal.
In France, Orkambi has been available to eligible patients ages 12 and older since December 2015 through a Temporary Use Authorization (Autorisations Temporaires d'Utilisation, or ATU) and a post-ATU interim access programme.
In addition to providing reimbursement for patients ages 12 and older, this agreement also enables people ages two to 11 years to access Orkambi in France for the first time.
All those who are currently prescribed Orkambi through the ATU will now receive it through this new agreement.
Vertex's CF medicines are reimbursed in 21 countries around the world, including recent agreements in Australia, England and Spain.
Cystic fibrosis is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract.
CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene.
Children must inherit two defective CFTR genes, one from each parent, to have CF.
While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation.
These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface.
The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs.
In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.
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.
Lumacaftor/ivacaftor 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 that invests in scientific innovation to create transformative medicines for people with serious diseases.
The company has four approved medicines in the US and three approved medicines in Europe that treat the underlying cause of cystic fibrosis, a rare, life-threatening genetic disease, and has several ongoing clinical and research programs in CF.
Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency, and APOL1-mediated kidney disease.
In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.
Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK.
Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America.
Vertex is consistently recognised as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes.