MPs to debate cystic fibrosis drug Orkambi in Parliament

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A debate is due to take place in Westminster about a drug for people living with cystic fibrosis to be made available on the NHS.

Cystic fibrosis, affects around 10,000 people in the UK and causes a sticky mucus to build up in the lungs and digestive system.

Campaigners want the precision drug Orkambi available to patients - but the UK Government is currently locked in negotiations over the cost of the drug with pharmaceutical firm Vertex.

The debate was triggered by a community-led petition that received over 100,000 signatures.

  • What is Orkambi?

Orkambi is one of the drugs which targets the root cause of the disease and would benefit around half of people with Cystic Fibrosis, according to the charity.

It has been licensed for use for more than three years, but a deal to make it available on the NHS for patients in England, Wales and Northern Ireland has still not been reached.

During that time, another drug to treat the root cause of the condition, Symkevi, has been approved for use but is also not available on the NHS.

David Ramsden, chief executive of the Cystic Fibrosis Trust, called it a

  • Why are negotiations still ongoing?

Orkambi has a list price of £104,000 per patient, per year and the drug must be taken for life.

A disagreement between the NHS and US firm Vertex Pharmaceuticals over the price of the drug still shows no signs of being resolved more than two years after negotiations started.

The NHS has offered £500 million, over five years, for access to Orkambi and other cystic fibrosis drugs made by Vertex, with the promise of renewals in the future.

But this has been rejected by Boston-based Vertex, which has secured deals with other countries for access to Orkambi and has criticised the way new drugs are appraised in the UK.

Vertex is understood to have offered to reduce this list price, though NICE has said the US company would need to drop the list price by 90% in order for it to be approved on the NHS.

Eight-year-old Sofia Bow has cystic fibrosis

Eight-year-old Sofia Bow has cystic fibrosis - and takes a variety of drugs every day.

Her parents say a row over Orkambi is depriving her of the treatment she needs.

In a statement, the Government said it "urges Vertex Pharmaceuticals to fully re-engage with the NICE appraisal process and to accept the offer the NHS made in July."

In March, a Welsh Government spokesman said: “We are guided by the recommendations of the National Institute for Health and Care Excellence (NICE) and the All Wales Medicines Strategy Group (AWMSG) about which medicines should be available on the NHS in Wales.