Six new medicines approved in Scotland including first treatment for rare heart condition
The Scottish Medicines Consortium (SMC) has approved Tafamidis for the treatment of a rare heart condition called amyloidosis cardiomyopathy (ATTR-CM). Pfizer UK said it was "an important milestone for eligible patients in Scotland".
Monday 13 November 2023 15:50, UK
Six new medicines have been approved for use in Scotland, including the first treatment for a rare "potentially life-threatening" heart condition.
The Scottish Medicines Consortium (SMC) has approved Tafamidis for the treatment of a rare heart condition called amyloidosis cardiomyopathy (ATTR-CM).
It is the first treatment to be licensed to treat wild-type or hereditary amyloidosis cardiomyopathy and Scotland is the first in the UK to make this medicine routinely available on the NHS, pharmaceutical company Pfizer said.
ATTR-CM is a rare condition where abnormal proteins called amyloid build up in tissues around the heart.
Dr Caroline Coats, consultant cardiologist at NHS Greater Glasgow and Clyde, said: "Transthyretin amyloid cardiomyopathy is a rare, under-diagnosed and potentially life-threatening condition, for which there is a significant unmet medical need.
"This decision is a welcome step to help expand treatment options for patients."
Owen Marks, head of rare diseases, Pfizer UK added: "This decision is an important milestone for eligible patients in Scotland, who until now have not had a specific treatment option for amyloidosis cardiomyopathy.
"We'll continue to work to help improve the care and diagnosis of ATTR-CM in Scotland and in the rest of the UK."
The SMC has also approved the use of selpercatinib on an interim basis to treat a rare type of non-small cell lung cancer.
It will be available to patients while further research is undertaken.
The SMC will review the evidence and make a decision on routine availability in NHS Scotland.
Durvalumab was approved for use to treat adults with advanced biliary tract cancer, which is cancer of the bile ducts that carry bile from the liver and the gallbladder to the intestines, which is newly diagnosed.
Avacopan was accepted for the treatment of adults with rare conditions that can cause blood vessels to become inflamed which can restrict blood flow and cause damage to vital organs.
Bimekizumab was accepted for the treatment of adults with psoriatic arthritis, an inflammatory arthritis associated with the skin condition psoriasis.
Risankizumab was accepted for treating Crohn's disease in patients aged 16 or older.
However, some medicines were not accepted for use.
The committee was unable to accept efgartigimod alfa for the treatment of adult patients with the muscle weakness condition generalised Myasthenia Gravis (gMG).
It was also unable to accept pegunigalsidase alfa for the treatment of Fabry disease, a rare inherited condition that can lead to kidney failure and heart problems.
Mercaptamine was also not accepted for use in patients with nephropathic cystinosis, a rare inherited condition that can affect the kidneys.
Read more from Sky News:
World's first whole-eye transplant hailed as breakthrough
Breast implants used in lifesaving surgery after lungs removed from man's body
Be the first to get Breaking News
Install the Sky News app for free
SMC chair Dr Scott Muir said: "We are very pleased to be able to accept six new medicines for use by NHS Scotland for a range of serious and disabling conditions.
"The evidence for the medicines we were unable to accept was not strong enough to satisfy the committee. We would welcome resubmissions for them once the companies have had an opportunity to address the key clinical and cost effectiveness uncertainties highlighted."