MHRA approves Alnylam's Amvuttra for rare heart disease ATTR-CM treatment

Alnylam Pharmaceuticals' innovative medication, Amvuttra (vutrisiran), has gained approval from the UK's Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of adults suffering from transthyretin amyloid cardiomyopathy (ATTR-CM). This rare condition affects the heart muscle and can lead to fatal outcomes. Amvuttra's new authorization covers both wild-type and hereditary forms of ATTR-CM, conditions caused by the buildup of misfolded transthyretin (TTR) protein. This accumulation can result in irreversible damage to the cardiovascular system and numerous severe health issues.

ATTR-CM can manifest in two main types. Wild-type ATTR-CM is usually linked with aging and is most frequently diagnosed in individuals over the age of 50. Hereditary ATTR-CM, on the other hand, is a genetic condition more commonly found in people of African descent. The MHRA's decision to approve Amvuttra was based on encouraging results from the HELIOS-B study, a late-stage clinical trial. In this study, Amvuttra demonstrated a notable ability to reduce mortality and cardiovascular events among patients suffering from ATTR-CM.

Beyond these benefits, Amvuttra has been shown to maintain the functional status and quality of life in patients. These positive effects were consistent across various patient subgroups, even in those who were also taking a TTR stabilizing medication. Marianna Fontana, an investigator involved in the HELIOS-B study, expressed optimism about the drug's potential to improve patient outcomes. She highlighted the significance of translating successful clinical trial results into practical treatment options that can benefit individuals affected by this swiftly progressing disease.

Administered as a subcutaneous injection every three months, Amvuttra can be given by healthcare professionals or self-administered by patients. The drug operates by leveraging the body's natural mechanisms to target and reduce TTR at its source, thereby slowing the accumulation of amyloid deposits. Prior to this new approval, Amvuttra was already authorized by the MHRA for treating hereditary TTR-mediated ATTR amyloidosis in adults with stage 1 or stage 2 polyneuropathy.

Phil Davey, Alnylam Pharmaceuticals’ country manager for the UK and Ireland, described the approval as a major advancement for individuals living with ATTR-CM and a source of great pride for the company. Since 2023, Amvuttra has been made available in the UK for patients with ATTR amyloidosis who exhibit polyneuropathy symptoms. This latest approval broadens the treatment scope, allowing the company to address the needs of a wider patient population affected by this condition.

Davey emphasized the company's commitment to ensuring that patients across the UK's National Health Service (NHS) can access this treatment without unnecessary delays. This collaborative effort aims to deliver timely healthcare solutions to those in need, reflecting Alnylam's focus on making significant strides in treating complex diseases like ATTR-CM.