Alnylam Pharmaceuticals’ Amvuttra (vutrisiran) has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) to treat adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a rare and potentially fatal disease of the heart muscle.
The drug has been authorised to treat wild-type or hereditary ATTR-CM, which occurs when misfolded transthyretin (TTR) protein accumulates and causes irreversible cardiovascular damage and severe health complications.
Wild-type ATTR-CM is associated with ageing and most commonly affects people aged over 50 years, while the hereditary form, which is passed down genetically, is more prevalent among people of African ancestry.
The UK regulator’s decision was supported by results from the late-stage HELIOS-B study, in which Amvuttra was associated with significant reductions in mortality and cardiovascular events in ATTR-CM patients.
Alnylam’s drug was also shown to preserve functional status and quality of life, and effects were found to be consistent across all patient subgroups, including those on a concomitant TTR stabiliser.
HELIOS-B investigator, Marianna Fontana, said: “As a physician, it’s a privilege to see a treatment that showed the potential to significantly improve outcomes in clinical trials now becoming available in practice – offering a new option for people living with this rapidly progressive disease.”
Given as a subcutaneous injection once every three months either by a healthcare professional or self-administered by patients, Amvuttra is designed to work with the body’s natural system to knock down TTR at its source, helping to slow the accumulation of amyloid deposits.
The drug is already approved by the MHRA to treat hereditary TTR-mediated ATTR amyloidosis in adults with stage 1 or stage 2 polyneuropathy.
Phil Davey, country manager, Alnylam Pharmaceuticals, UK and Ireland, said: “[This] announcement is a significant step forward for people living with ATTR-CM and a moment of immense pride for Alnylam.
“Since 2023, [Amvuttra] has been available in the UK for ATTR amyloidosis patients with polyneuropathy symptoms, and this expanded indication enables us to address the needs of a much broader group of people living with the disease.”
Davey added that the company will now focus on “working collaboratively to secure access for patients across the NHS, without delay”.