It has been confirmed that the HSE will fund a new drug used to treat a rare condition known as the ‘Donegal disease.’
Amyloidosis is caused by deposits of abnormal protein – amyloid – in tissues and organs throughout the body.
One variant of ATTR amyloidosis, known as ATTR T60A (or Thr60AIa), was first identified in an Irish family in 1986. A cluster of cases have since been identified in County Donegal, where up to 1 per cent of the population are estimated to possess the mutation. The North’s former Deputy First Minister, Martin McGuinness, died from the condition in May 2017. Mr McGuinness’ late mother was from Donegal and he spent much of his time in Buncrana.
Today the manufacturer of the drug Vutrisiran , Alnylam Pharmaceuticals, confirmed the HSE will fund the medication for users in Ireland as a treatment for adults with the hereditary disease. From 22 May, the treatment has been available in accordance with a Managed Accessed Protocol (MAP). Vutrisiran is an RNAi medicine that targets the underlying cause of the disease. It works by lowering the production of the protein responsible for the build-up of amyloid, ultimately acting to reduce the effects of this illness.
Amyloid deposits can accumulate in various organs and tissues in the body, including the nerves, which may involve a loss of sensation in the lower limbs, hands and loss of mobility, the impact can be particularly severe. If left untreated, the condition can be life-threatening.
Derry woman Rosaline Callaghan, Founder at Amyloidosis Ireland, commented: “While hATTR amyloidosis is considered a rare condition, its impact on patients and their families should not be underestimated. Throughout Ireland, and particularly in the North-West, there is a prevalent genetic variant at the root of this condition. Today’s announcement offers Irish patients more options.”
Phil Davey, Country Manager, Alnylam Pharmaceuticals, UK and Ireland, said: “We are delighted with the decision from the Health Service Executive to approve reimbursement for vutrisiran for patients living with hATTR amyloidosis with stage 1 or stage 2 polyneuropathy. Our commitment is to ensure that all eligible patients who could benefit from our medicines are able to access them without delay, and this marks another important step forward on that journey.”
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