A WONDER drug which has been approved for use in Scotland for people with cystic fibrosis has been hailed as life-saving.
The new triple therapy treatment will be available to cystic fibrosis patients on the NHS, following an agreement between the Scottish Government, NHS Scotland and the pharmaceutical company behind it.
And there are around 900 Scots with the lung condition and many of them could benefit from the treatment.
It had been approved for use in England in June and campaigners were hopeful it would be given the go ahead north of the border as well. Those living with the genetic lung condition welcomed yesterday’s announcement saying it will change lives.
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David Ramsden, Chief Executive of the Cystic Fibrosis Trust, said: “It’s fantastic that the Scottish Government and Vertex Pharmaceuticals have agreed a deal on Kaftrio. Available to people across Scotland in the coming weeks, this new drug will truly save lives.
“While this is a special moment for people with cystic fibrosis in the four nations, there is more to do and we will not stop until everyone with cystic fibrosis across the UK has access to life-saving drugs.”
Shona Young, 25, from Glasgow, who has cystic fibrosis, said this is the news she had been waiting for. It comes just days after shielding was paused for those who may have been more susceptible to coronavirus and she will also be returning to the classroom assistant job she loves on Monday.
Ms Young said: “Because of Kaftrio, my whole life is going to change. I will, for the first time, be able to plan ahead for a longer future. I’m overwhelmed and am so excited about the new possibilities this treatment will open up for me and for so many others with cystic fibrosis. Kaftrio is what we have all been waiting for and I couldn’t be more grateful that it exists.”
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Ms Young has to take a daily cocktail of drugs and undergo physiotherapy to help move the build of mucus in her lungs. She is also prone two chest infections and can be hospitalised for further treatment.
She added: “It has been a waiting game. England had approved, followed by Wales and Northern Ireland so there was a lot of anticipation. It really is the biggest news for the Cystic Fibrosis community. It will stop my lungs getting infected so often and make my general health so much better. It will mean less hospital admissions as well and less time off work.
“It’s the little things this might help such as not feeling so ill or waking up feeling tired. This will allow me to live as much of a normal life as I could. I might be able to exercise more and maybe even take up a new hobby without feeling so tired.”
Kaftrio, produced by Vertex Pharmaceuticals, is a medicine that tackles the underlying causes of the disease by helping the lungs work more effectively.
Health Secretary Jeane Freeman, said she was delighted that an agreement had been reached for the treatment to be available.
Ms Freeman said: “I am therefore very pleased that a revised pricing agreement has been reached between NHS National Procurement and Vertex Pharmaceuticals, to ensure that Kaftrio, can be made available for the treatment of cystic fibrosis through the NHS.
“This is a potentially transformational treatment for cystic fibrosis, and I am delighted that patients in Scotland will be among the first in Europe to benefit and lead longer, healthier lives.
“The deal, agreed ahead of a licence being granted by the European Medicines Agency, means that clinicians working in cystic fibrosis treatment centres, will be able to prescribe the medicine to patients whom they consider will benefit from their use as soon as the licence is granted, in the coming weeks. I hope that this news will be welcome to many people who live with cystic fibrosis, and also their families and friends.”
There are an estimated 800 people with cystic fibrosis in Scotland should be suitable for treatment with the range of medicines that are now available. The terms of the deal also mean that many patients with rare mutations, which fall outside of the scope of the European Medicines Agency’s current licensing considerations, will also be able to benefit from Kaftrio.
A spokesman for Vertex said: "We look forward to continue working in collaboration with the authorities in Scotland to complete all necessary steps and finalise the agreement as soon as possible."
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