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.
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“This decision is a welcome step to help expand treatment options for patients.”
Joel Rose, chief executive of Cardiomyopathy UK, said: “Amyloidosis cardiomyopathy significantly impacts everyday life for an individual and progressively worsens over time.
“Therefore, we’re pleased there is now an approved treatment option for eligible patients living in Scotland which could help improve their care.”
Owen Marks, head of rare diseases, Pfizer UK said: “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 which 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 called with severe active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA).
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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.
SMC chair Dr Scott Muir said: “We are very pleased to be able to accept six new medicines for use by NHSScotland for a range of serious and disabling conditions.
“The early data for selpercatinib in a form of lung cancer is very promising. There will be a further review of updated evidence to ensure that this treatment offers what is expected in terms of clinical outcomes and good value for patients in NHS Scotland.
“Tafamidis is the first treatment to be licensed for transthyretin amyloidosis cardiomyopathy. The company presented data that suggested there would be improvements in mortality and fewer hospitalisations for patients with this terrible condition.
“From the evidence given by patients and clinicians, we know that advanced biliary tract cancer has a very poor prognosis. Adding durvalumab to current treatment may increase survival for some patients, so we know this decision will be welcomed.
“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.”
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