New drug ‘landmark moment for people with cystic fibrosis’ says Dundee teen Adam

September 14, 2019, 6:06 am

Hundreds of cystic fibrosis patients across Scotland could finally get the chance to try a potentially life-changing drug now available on the NHS.

The Scottish Government has announced that Orkambi and Symkevi will be offered to people with particular strands of CF, thanks to a five-year agreement with drug manufacturer Vertex Pharmaceuticals and support from the UK CF Registry.

Just last month the Scottish Medicines Consortium (SMC) had denied the use of the drugs on the NHS, citing uncertainty around the long-term benefits coupled with the prohibitive cost of £100,000 a year per patient.

Earlier this year the Cystic Fibrosis Trust held a space hopper bounce across the Tay Road Bridge to raise funds for the charity.

The deal now requires Vertex to resubmit new data collected through NHS prescribing to the SMC during the agreement period.

Cystic fibrosis is a life-shortening genetic condition where the lungs and digestive system become clogged with sticky mucus, making it difficult to breathe and properly digest food.

There are currently around 350 people in Scotland who could benefit from the drugs, including Dundee teenager Adam Lyon.

Adam, 19, from Fairmuir, who has the dd508 mutation, said of the news: “It’s brilliant, it’s amazing.

“If there is something out there that is meant to be able to dramatically help people with cystic fibrosis then it’s fantastic it’s being made available. It’s great news overall.

“I don’t know how quickly it will happen. I’m looking forward to hopefully feeling its use.”

Adam’s mum Linda said: “It’s fantastic news. Fingers crossed it might help some of our children live longer.”

Morag Beckett, who runs the Cystic Fibrosis Tayside Group and whose son Greg could benefit from Orkambi, said: “As a family we are cautiously optimistic. I won’t quite believe it until it is in our medicine cupboard.

“I feel that Greg and others with CF deserve this, they have been promised this for so long. I can’t quite believe that it’s happening.

Morag Beckett, Greg Beckett with friend Laura Ross.

“I’m sad that for so many it has come too late.

“I’m just delighted that many of those who live with cystic fibrosis now have a better chance of a longer life and will be able to fulfil many of their dreams.

“For too long many have suffered way more than any young person should.”

Greg Beckett said: “I’m obviously really excited, a lot of people put a lot of effort into raising funds and awareness so its great to see that paying off, hopefully it wont be long before the drug makes its way to those who can benefit from it.”

David Ramsden, chief executive of the Cystic Fibrosis Trust, said: “This is a landmark moment for the hundreds of people with cystic fibrosis and their families across Scotland who have tirelessly campaigned for years to access these drugs. This breakthrough is a victory for their perseverance and enduring hope.

“It means 350 eligible people living in Scotland will have access to drugs that stabilise their lung health and reduce the need for hospital admissions.