Vertex Pharmaceuticals Incorporated, a global biotechnology company, has reached a pricing and reimbursement agreement for Orkambi (lumacaftor/ivacaftor) with the German Federal Association of the Statutory Health Insurances (GKV-SV).

Orkambi is the first medicine to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation in the CF transmembrane conductance regulator (CFTR) gene. CF is a rare and life-shortening genetic disease caused by a defective or missing CFTR protein resulting from a mutation in the CFTR gene. The disease is present from birth and causes chronic lung infections and progressive damage to a number of organs throughout the body.

Today's announcement follows a comprehensive benefit assessment of the medicine by the German Federal Joint Committee (G-BA), which recognized the "considerable additional benefit" of Orkambi for people with CF who have two copies of the F508del mutation. Orkambi has been available to eligible patients in Germany since it was granted marketing authorization from the European Commission in November 2015, and the reimbursement agreement announced today took effect on December 16, 2016.

"Cystic fibrosis is a progressive disease, meaning that with each passing day, lung function can deteriorate and patients get sicker," said David Gillen, M.D., vice president of international medical Affairs at Vertex. "We are pleased the German authorities approved Orkambi reimbursement for all eligible patients. The reimbursed price recognizes the clinical value of Orkambi and the need for Vertex's continued investment in the research and development of new medicines for the two out of three people with CF still waiting for a treatment for the underlying cause of their form of the disease."

In addition to Germany, Orkambi is now available to eligible people with CF in the United States, Austria, and France. Vertex remains actively involved in reimbursement discussions in many other countries to make Orkambi available to all people who can benefit from this important medicine. Earlier this month, Orkambi received the "Drug Discovery of the Year" award from the British Pharmacological Society and the French "Prix Galien" award for the most promising rare disease medicine in 2016.

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 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. Orkambi is available as tablets and is typically taken twice per day.