Treating cystic fibrosis

A 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.

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:

“Cystic fibrosis is an inherited condition, which tragically shortens lives and affects around 900 people in Scotland. 

“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.”

Background

It is estimated that around 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.