By Neil Trainis

The Scottish Medicines Consortium (SMC) has today approved six new medicines including a treatment for a rare heart condition, a decision its manufacturer Pfizer described as “an important milestone” for patients in Scotland . 

Tafamidis was approved for amyloidosis cardiomyopathy (ATTR-CM), a potentially fatal and often undiagnosed condition in which a protein called transthyretin which normally circulates in the bloodstream builds up in the heart, nerves and other organs.

It is believed to be the first licensed treatment for wild-type or hereditary ATTR-CM and Scotland is the first UK nation to make it available on the NHS.

Dr Caroline Coats, consultant cardiologist at NHS Greater Glasgow and Clyde, told Sky News : “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.”

The SMC also approved selpercatinib for the treatment of a rare type of non-small cell lung cancer and although more research is being carried out to decide if it will be routinely made available on the NHS, patients can currently access it on an interim basis.

Durvalumab was approved to treat adults with advanced biliary tract cancer, Avacopan was passed for adults with rare Conditions that cause blood vessels to become inflamed, Bimekizumab was approved for adults with psoriatic arthritis and Risankizumab was approved for the treatment of Crohn's disease in patients aged 16 and above.

However, three medicines - efgartigimod alfa for the treatment of adults with Myasthenia Gravis, a rare long-term condition that causes muscle weakness, pegunigalsidase alfa for the treatment of Fabry disease, an inherited neurological disorder that can lead to kidney failure and heart problems, and Mercaptamine for the treatment of nephropathic cystinosis, were not approved by the SMC.

Its chair Dr Scott Muir said it was unable to approve those medicines because the evidence “was not strong enough” but encouraged their manufacturers to resubmit them after they have addressed “the key clinical and cost-effectiveness uncertainties highlighted.”