Vertex Pharmaceuticals announced that it has expanded its reimbursement agreement with NHS England for Vertex’s cystic fibrosis medicines to include Kaftrio (ivacaftor/tezacaftor/elexacaftor), in a combination regimen with Kalydeco (ivacaftor) 150 mg, ahead of the medicine’s anticipated approval by the European Commission (EC).
The new expanded agreement includes reimbursed access to Vertex’s currently licensed medicines — Kalydeco (ivacaftor), Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor), as well as the triple combination therapy if approved — and any future additional licensed indications for all of these medicines.
Reshma Kewalramani, M.D., chief executive officer and president at Vertex, said, “I’m pleased that NHS England has recognized the value of Kaftrio, and that Vertex and NHS England have been able to work quickly, collaboratively and flexibly to expand the existing reimbursement agreement to include the triple combination therapy in advance of the medicine being licenced. This will ensure that eligible patients in England will be among the first in Europe to benefit from access to this innovative medicine upon approval.”
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recently adopted a positive opinion for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with Kalydeco (ivacaftor) 150 mg to treat people with cystic fibrosis (CF) ages 12 and older with one F508del mutation and one minimal function mutation (F/MF) or two F508del mutations (F/F) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
As part of the agreement with NHS England, Vertex has committed to submit Orkambi, Symkevi and Kaftrio to the National Institute for Health and Care Excellence (NICE) within an agreed upon timetable, allowing for a period of real-world data collection on the medicines.
Vertex will be working closely with the authorities in Northern Ireland, Wales and Scotland with the aim of securing an equivalent agreement in those countries as soon as possible.
Cystic fibrosis (CF) 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.
Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with Kalydeco (ivacaftor) is an investigational medicine developed for the treatment of cystic fibrosis (CF) in patients ages 12 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. It is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface. It recently received a CHMP positive opinion, with the EU license expected over the next few months.