Alnylam’s RNAi therapeutic recommended by NICE for hereditary form of amyloidosis

Alnylam Pharmaceuticals’ Amvuttra (vutrisiran) has been recommended by the National Institute for Health and Care Excellence (NICE) as an option for treating a hereditary form of amyloidosis, the RNA interference (RNAi) therapeutics company announced.

The decision from NICE makes Amvuttra one of the first rare disease medicines to receive a positive draft recommendation under its streamlined pilot process that aims to accelerate patient access to promising new treatments.

Following final guidance, patients with hereditary transthyretin-related (ATTR) amyloidosis in England will have the option of Amvuttra, which is given as a subcutaneous injection every three months, compared to existing therapies that need to be administered every few weeks or more frequently.

The guidance is expected to be implemented in Wales in due course, the company said.

ATTR amyloidosis is a rare and serious disease caused by one of the proteins that is made by the body not being produced correctly. The protein, which is called transthyretin (TTR), can become abnormal in ATTR amyloidosis and break apart into smaller pieces (amyloid).

These deposits can build up in different organs and tissues in the body, such as the heart, eyes, nerves and gastrointestinal tract, resulting in damage and creating potentially life-threatening health problems.

When left untreated, patients with ATTR amyloidosis can develop polyneuropathy – disease of the peripheral nerves – which can result in a loss of sensation in the lower limbs and hands, autonomic dysfunction and a loss of mobility.

Amvuttra is an RNAi therapeutic that seeks to address the root cause of ATTRv amyloidosis by inhibiting the production of the TTR protein by the liver, leading to a reduction in the level of TTR in the blood.

The company’s NICE application was supported by data from the HELIOS-A study in which treatment with Amvuttra demonstrated TTR reduction of 84.7% at month 18 versus 80.6% for standard of care Onpattro (patisiran), Alnylam’s earlier RNAi ATTRv amyloidosis therapy that is administered intravenously every few weeks.

Professor Julian Gillmore, consultant at the UCL National Amyloidosis Centre, at the Royal Free Hospital, London said: “Today’s recommendation of [Amvuttra] marks another remarkable step forward for patients with hereditary ATTR amyloidosis.

“This medicine targets the production of amyloid at the source – an approach that we already know can be highly effective at slowing down the symptoms of this condition. Now, we have the ability to do this with an injection that can be given every few months, rather than every other week.