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SMC accepts AMVUTTRA®▼ (vutrisiran) as a treatment for hereditary form of amyloidosis on the NHS in Scotland

MAIDENHEAD, UK, 11th September 2023 – Alnylam UK Limited, the leading RNA interference (RNAi) therapeutics company, today announced that, following an abbreviated submission, the Scottish Medicines Consortium (SMC) has accepted AMVUTTRA® (vutrisiran) for use in NHS Scotland as a treatment for adults with hereditary transthyretin-mediated (ATTRv) amyloidosis with stage 1 or stage 2 polyneuropathy.[i]

AMVUTTRA is an RNA interference (RNAi) therapeutic that is administered via subcutaneous injection once every three months. It aims to target the root cause of ATTRv amyloidosis, by inhibiting the production of the transthyretin (TTR) protein. TTR is the cause of progression in this rare, progressive disease that can be fatal if left untreated.ii 

Vince Nicholas, an ATTRv patient and Trustee at the UK ATTR Amyloidosis Patients’ Association (UKATPA), said: “ATTRv amyloidosis is a disease that can be extremely debilitating, and frequent hospital or scheduled nurse visits to receive treatment can further diminish a person’s independence and ability to live life to the fullest. Today’s news is a major milestone for patients in Scotland, who will now have the opportunity to receive a treatment that only needs to be given once every three months, allowing them more time to spend doing what matters most to them.”

ATTRv amyloidosis is a hereditary condition where one of the proteins that is made by the body, TTR, is produced abnormally. The TTR protein occurs naturally, being produced in the liver and coded for by the TTR gene. In cases of ATTRv amyloidosis, TTR is not produced correctly. As a result, it can break apart into smaller pieces, known as amyloid, that can deposit and accumulate in different organs and tissues in the body, such as the nerves, heart, eyes and gastrointestinal tract. Left untreated, resultant tissue damage can lead to numerous health issues. This includes polyneuropathy (disease of the peripheral nerves), which is associated with a loss of sensation in the lower limbs and hands, and loss of mobility. Additionally, patients could experience autonomic dysfunction, affecting various organs such as the heart, bladder, and intestines, among others.i[ii]

Vutrisiran works by inhibiting the liver’s production of the TTR protein, resulting in lower levels of TTR in the blood. This prevents the build-up of amyloid deposits and thereby halts the progression of ATTRv amyloidosis. By doing so, vutrisiran can make it possible to slow or stop the course of the disease, limiting further damage to patients’ organs and bodily tissues.

Phil Davey, Country Manager, Alnylam Pharmaceuticals, UK and Ireland, said: “Today’s approval of vutrisiran is positive news for patients living with ATTRv amyloidosis in Scotland. This rapid review and recognition of vutrisiran in Scotland builds on the medicine’s reimbursement from NICE for England and Wales earlier this year, reinforcing our mission to ensure that all patients can access potentially life-saving treatments.”

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Last Updated: 19-Sep-2023