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Scotland approves six new medicines including Tafamidis for treatment of rare heart condition


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Pfizer data suggested that their drug would lead to improvements in ‘mortality and fewer hospitalisations’ for patients with this terrible condition 

The Scottish Medicines Consortium (SMC) has approved six new medicines including the first treatment for a “potentially life-threatening” heart disease.

Transthyretin amyloidosis cardiomyopathy (ATTR-CM) is a rare heart condition where abnormal proteins called amyloid build up in tissues around the heart, nerves, and other organs of the body.

Dr Scott Muir, the SMC Chair, 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.

Tafamadis 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.”

Pfizer, the manufacturer of the approved drug, has described it as “an important milestone” for eligible patients in Scotland” who until now have not had a specific treatment option for the disease.

Owen Marks, the head of rare diseases at Pfizer UK, told Sky News that they will continue to work to help “improve the care and diagnosis of amyloidosis cardiomyopathy in Scotland and in the rest of the UK.”

Dr Caroline Coats, a consultant cardiologist at NHS Greater Glasgow and Clyde told the publication that there is a significant unmet medical need for transthyretin amyloid cardiomyopathy.

The SMC’s decision is a welcome step to help expand treatment options for patients, she added.

Five other new drugs approved in Scotland

Selpercatinib was approved on an interim basis for the treatment of a rare type of non-small cell lung cancer.

The medicine will be available to patients while further information is gathered, and the SMC will decide on its routine availability in NHS Scotland after reviewing the evidence.

Dr Muir said: “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 NHSScotland.”

Durvalumab got the SMC’s nod for the treatment of adults with advanced biliary tract cancer.

It is a cancer of the bile ducts which carry bile from the liver and the gallbladder to the intestines, which is newly diagnosed.

Dr Muir commented: “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.”

Avacopan is the new treatment approved for use in adults with severe active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA).

These are rare conditions that cause blood vessels to become inflamed which can restrict blood flow and cause damage to vital organs such as the lungs and kidneys.

The SMC has also approved Bimekizumab for the treatment of adults with psoriatic arthritis, an inflammatory arthritis associated with the skin condition psoriasis.

Risankizumab was licensed for treating Crohn’s disease in patients 16 years or older.

The committee, which advises on newly-licensed medicines for use by NHS Scotland, didn’t accept three new medicines.

It was unable to accept Efgartigimod alfa for the treatment of adult patients with generalised Myasthenia Gravis (gMG)

The committee also didn’t approve Pegunigalsidase alfa for the treatment of Fabry Disease and Mercaptamine for use in patients with nephropathic cystinosis.

“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,” the SMC Chair added.


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