Now 1,000 days since cystic fibrosis drug Orkambi was licensed in the UK, but it’s still denied to the British public

– Wednesday, 15^th August – marks 1,000 days since the cystic fibrosis medicine Orkambi was licensed in the UK – but it is still unavailable on the NHS.

During that time, more than 200 people who may have benefited from this life enhancing and potentially life prolonging drug have sadly died.

Cystic fibrosis is a terrible and debilitating life-shortening illness that leads to a severely reduced quality of life.

It causes a build-up of thick, sticky mucus, chronic lung infections and progressive lung damage.

Daily life itself can be a struggle and people with the condition face a huge burden of daily treatments.

Orkambi is a precision medicine that nearly half of the more than 10,000 people in the UK with cystic fibrosis, including children aged six or over, could benefit from.

While conventional cystic fibrosis treatments target the symptoms, precision medicines such as Orkambi tackle the underlying genetic defects that cause the condition.

Orkambi has been found to slow decline in lung function by up to 42 per cent – the most common cause of death for people with cystic fibrosis.

It has also been shown to reduce chest infections requiring hospital treatment by up to 61 per cent.

Orkambi received its European license on 20 November 2015 and in June the following year the UK government agency the National Institute for Health and Care Excellence (NICE) recognised Orkambi as an important treatment.

Yet despite this it is unavailable to most people with cystic fibrosis across the UK. And today it is 1,000 days since it was licensed for use in Europe and therefore the UK.

Earlier this year, MPs debated making Orkambi available while hundreds of children wrote personal, heartfelt letters to the Prime Minister pleading for it to be made available

And when asked about the issue during Prime Minister’s Questions, Theresa May said she wanted to see a quick resolution to the negotiations to ensure people can access the drugs.

However, over the summer talks between the drugs manufacturer Vertex and NHS England broke down, shattering the hopes of thousands of people.

MP Ian Austin, Member for Dudley North who has ardently campaigned to make Orkambi available in the UK, said: “It is extremely disappointing that medicines such as Orkambi are still not available on the NHS, even though they have been available to patients the USA and several European nations for over two years.

“I’ve met people who have their lives changed by this drug and I’ve met many more who are desperate to be given it.

“It’s crucial now that the Government and pharmaceutical companies come to a speedy and positive resolution so that people with Cystic Fibrosis in the UK can get the support they urgently need and I will be sure to keep pushing them on this issue.”

Lynsey Beswick, Public Affairs Manager at the Cystic Fibrosis Trust, said: “It is extremely disappointing that while new medicines are being made that can really change and potentially prolong the lives of people with cystic fibrosis they are being denied to people in the UK.

“Theresa May recently said she would like to see a speedy resolution to this issue, but still we seem no closer to achieving this.

“We urge the NHS and drug manufacturer Vertex to agree a resolution that will enable speedy access. This wait is simply unacceptable. People with CF have waited far too long.”