EC Approves Alnylam's Amvuttra for Treating Rare Heart Disease ATTR-CM

Alnylam Pharmaceuticals has received approval from the European Commission for its RNAi therapeutic, Amvuttra (vutrisiran), to treat transthyretin amyloid cardiomyopathy (ATTR-CM) in adults. This heart muscle disease, which can lead to severe health issues, affects both hereditary and wild-type ATTR-CM patients. ATTR-CM is caused by the accumulation of misfolded transthyretin (TTR) proteins, leading to progressive cardiovascular damage. While hereditary ATTR-CM impacts about 50,000 individuals globally, wild-type ATTR-CM affects up to 300,000 people. The disease may present with symptoms of cardiomyopathy, polyneuropathy, or both.

Amvuttra is administered through a subcutaneous injection every three months. It functions by reducing TTR protein levels at the source, aligning with the body's natural processes. Previously, this treatment had been approved in the EU for managing hereditary TTR-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy. Pushkal Garg, Alnylam’s Chief Medical Officer, highlighted the significance of Amvuttra’s approval, stating that its ability to provide rapid and sustained TTR reduction through quarterly doses offers a unique clinical approach, potentially transforming patient outcomes dealing with this challenging condition. Garg expressed the company's commitment to ensuring swift access to Amvuttra for eligible patients across the EU.

The European Commission's approval followed a positive recommendation from the European Medicines Agency’s human medicines committee, which was based on the outcomes of the HELIOS-B trial, a late-stage study. The trial evaluated Amvuttra both as a standalone treatment and in combination with standard therapies, such as tafamidis and SGLT2 inhibitors. The drug demonstrated a 28% reduction in the risk of all-cause mortality and recurrent cardiovascular events compared to a placebo. Furthermore, there was a 36% reduction in mortality observed at 42 months in a pre-specified secondary endpoint analysis, which included up to 36 months of data from the double-blind period and an additional six months from an open-label extension.

Marianna Fontana, an investigator for the HELIOS-B trial, emphasized the importance of these findings. She noted that the trial's diverse participant pool was representative of real-world clinical scenarios, enhancing the relevance of its results. Fontana described the approval of Amvuttra as a significant milestone for patients, providing a new treatment pathway that could considerably enhance disease outcomes.

This recent approval marks a progressive step in the treatment landscape of ATTR-CM, offering hope to many who suffer from this debilitating condition. By targeting the underlying cause of the disease, Amvuttra represents a noteworthy advancement in therapeutic options, enabling better management of the disease's cardiovascular implications. As the company moves forward, the focus will be on making this treatment accessible to patients across Europe, potentially improving quality of life and health outcomes for many affected by ATTR-CM.