Access to Orkambi and other drugs for people with cystic fibrosis

Orkambi is the brand name of a medicine (lumacaftor–ivacaftor) that is licenced to treat cystic fibrosis in patients who have a specific genetic mutation causing the disease (called the F508del mutation).   The Cystic Fibrosis Trust describes how this medicine works:

Orkambi is a combination medicine, made up of ivacaftor and lumacaftor. Lumacaftor helps get more proteins to the surface of cells in the body, and ivacaftor helps the chloride channels in the cells to operate more effectively. The combination of these two things helps to keep a healthy balance of salt and water in the organs – particularly the lungs.^[1]

In 2016, the National Institute for Health and Care Excellence (NICE) reviewed the use of Orkambi and concluded that it could not recommend it for routine use on the basis that it could not be considered cost-effective.^[1]

Since this time, there have been ongoing discussions between the pharmaceutical company that makes Orkambi, Vertex, and NHS England regarding the funding of this medicine.

The initial licence was for use only in patients aged 12 and over, but in January 2018, the manufacturer, Vertex, announced that the EU licence for this medicine has been extended for use in children aged 6 to 11 years.^[1] Orkambi has subsequently been licensed for 2 to 5 year olds in January 2019.^[3]

There has been ongoing campaigning for access to Orkambi. A petition on the Government and Parliament Petitions website calls on the Government to conclude the negotiations with vertex and fund the drug. At the time of writing, it has been signed by over 108,000 people. The Petitions Committee have tabled a debate on the petition for 10 June 2019. The debate will be led by Paul Scully MP.

A debate on a similar petition was held in March 2018.^[4]

[1]     Cystic Fibrosis Trust, Orkambi

[2]     NICE, Lumacaftor–ivacaftor for treating cystic fibrosis homozygous for the F508del mutation, July 2016

[3]     Vertex, Vertex Receives Expanded EU Approval for ORKAMBI® (lumacaftor/ivacaftor) in Children Ages 6-11 Years, January 2018

[4]     HC Deb 19 March 2018 C1 WH