13-year-old's plea for 'life-changing' cystic fibrosis drug Orkambi to be made available on the NHS

Amalie Pledger,who has cystic fibrosis, received the 'life-saving' drug Orkambi on compassionate grounds. Credit: ITV News
  • By Nitya Rajan: ITV News producer

Thousands of people with cystic fibrosis who have been denied the 'life-saving' drug Orkambi have seen their petition gain more than 100,000 signatures ahead of a debate in the House of Commons.

Campaigners have told ITV News the drug is "life-changing" and should be available on the NHS.

People with cystic fibrosis have a life expectancy between 30 to 40 years and while Orkambi is not seen as a cure, it is said to slow the decline of lung function, which is the most common cause of death for people with the condition.

Amalie Pledger, 13, is currently using Orkambi after being placed on Vertex Pharmaceutical's compassionate use programme.

Describing how the drug has changed her life, Amalie told ITV News: "Eventually I stopped planning where I was going to walk between lessons and stopped asking my friends to go slow."

Currently there is a deadlock between the drug's manufacturers, Vertex Pharmaceuticals, and the Department of Health.

Cavan Arrowsmith, a campaigner who has cystic fibrosis is among those urging the government to reach a compromise.

He told ITV News: "We need this to be able to stop progression of the disease, being able to live past the age of 38 and to be honest at the age of 31 potentially being faced with death in seven years really scares me."

Health watchdog NICE has recognised Orkambi as an "important treatment" but its cost has proved to be a barrier in making it available on the NHS.

Labour MP Ian Austin, who supports the campaigners' call for a compromise, told ITV News: "NICE have approved the drug for use, they say it's effective but they say it's not cost-effective."

Unlike most drugs on the market, Orkambi's effectiveness lies with its ability to treat a mutation known as F508del, which around 50% of people with cystic fibrosis in the UK have, instead of the symptoms.

ITV News has reached out to the government for comment.