Alnylam's ATTR cardiomyopathy drug backed for NHS use

Patients in England and Wales with cardiomyopathy caused by the disorder ATTR amyloidosis will soon be able to access treatment with Alnylam's gene-silencing drug Amvuttra.

The ruling by reimbursement authority NICE means that Amvuttra (vutrisiran) can be used to treat wild-type or hereditary ATTR amyloidosis with cardiomyopathy (ATTR-CM) in adults, two years after it was cleared for use by the NHS to treat polyneuropathy associated with the disorder (ATTR-PN).

The new recommendation comes just a few months after the UK drugs regulator, the MHRA, expanded the label for Amvuttra to include ATTR-CM, a progressive and ultimately life-shortening condition caused by the buildup of misfolded transthyretin proteins in cardiac tissue, leading to thickening and stiffening of the ventricles.

There are about 1,500 patients in the UK who may be eligible for treatment with the drug, according to Alnylam.

Amvuttra is a follow-up to Alnylam's earlier ATTR amyloidosis therapy Onpattro (patisiran), which was cleared by NICE for NHS use in 2019, but offers a simpler, more patient-friendly dosing regimen. It is administered as a subcutaneous injection every three months, while Onpattro is dosed intravenously every few weeks.

The newer drug has become Alnylam's top-selling medicine, with global sales rising 162% to $685 million in the third quarter of this year, driven by the new ATTR-CM indication, with Onpattro shrinking to below $40 million.

Alnylam's main rival in the market is Pfizer, whose orally-administered Vyndaqel (tafamidis) is already used by the NHS for ATTR-CM, having been recommended by NICE last year, three years after being turned down for being too expensive.

NICE's guidance document recommends that the NHS "use the least expensive option of the suitable treatments," including Amvuttra and Vyndaqel, after discussing the advantages and disadvantages of the available treatments with the person living with the condition.

It cites indirect clinical comparisons of the two drugs that suggest the two drugs are equally effective at extending life and delaying the worsening of cardiomyopathy. Amvuttra has a list price of almost £96,000 ($125,000) per pre-filled syringe, but will be supplied to the NHS at a confidential discount.

Earlier this month, Alnylam reported new data from the phase 3 HELIOS-B trial of Amvuttra in ATTR-CM, which showed that the drug reduced the risk of the composite of all-cause mortality or first cardiovascular event by up to 42% compared to placebo over four years.

Anna Tomlinson, chief executive of Cardiomyopathy UK, said NIVE's recommendation is "an important milestone for those living with ATTR-CM and their families" and "a step towards wider improvements—better diagnostic pathways, stronger access to specialist services, and the support families need to navigate such a complex condition."

She added that further work is needed to "ensure equitable access across all regions." So far, the Scottish Medicines Consortium (SMC), NICE's counterpart north of the border, has not delivered a decision on Amvuttra in ATTR-CM.