After months of talks, NHS England has recently reached a deal with Vertex Pharmaceuticals Incorporated (Vertex) for all currently licensed Vertex cystic fibrosis medicines and any future indications of these medicines.

The deal includes the drug Orkambi, a combination of lumacaftor and ivacaftor which is indicated for cystic fibrosis patients age 2 years and older who are homozygous for the F508del mutation. As reported in our earlier article, an offer by NHS England in July 2018 of £500 million over five years for all of Vertex’s cystic fibrosis drugs was rejected by Vertex.

This deal will come as huge relief for affected cystic fibrosis patients and their families, who have long campaigned for Orkambi to be made available on the NHS.

Cystic fibrosis (CF) is a life-shortening genetic disorder caused by the presence of mutations in both copies of the gene for the CF transmembrane conductance regulator (CFTR) protein. This protein functions as a channel across the membrane of cells that produce mucus, saliva, tears, sweat and digestive enzymes. The channel transports chloride ions (amongst others) into and out of cells, and thus helps to control the movement of water in tissues, which is necessary for the production of freely flowing mucus.

Mutations in the CFTR gene alter the production, structure or stability of the channel. This in turn impairs the transport of chloride ions and the associated movement of water. As a result, the mucus produced by cells that line the passageways of the lungs, pancreas and other organs is abnormally thick. This abnormal mucus obstructs the airways and glands.

More than 1,000 mutations in the CFTR gene have been identified in people with cystic fibrosis. Most of these mutations result in a single amino acid change or deletion. The most common mutation in the CFTR gene is a deletion of one amino acid at position 508 in the CFTR protein (the F508delta mutation).

NHS England’s deal means cystic fibrosis patients aged 2 years and older who have two copies of the F508del mutation in the CFTR gene can be prescribed Orkambi. The agreement also offers expanded access to Vertex cystic fibrosis drugs Symkevi and Kalydeco for use by eligible patients.

Details of the deal, including how much NHS England will pay Vertex were commercially confidential. However, announcing the agreement, NHS Chief Executive Simon Stevens said “I’m pleased that Vertex has now agreed a deal that is good for our patients and fair to British taxpayers.”