CHMP Recommends Alnylam's RNAi Drug Vutrisiran for Rare Heart Disease

Alnylam Pharmaceuticals has made a significant step forward with its RNAi therapeutic, vutrisiran, as it receives a recommendation from the European Medicines Agency's human medicines committee to treat adults with transthyretin amyloid cardiomyopathy (ATTR-CM). This condition is a severe heart muscle disease resulting from the accumulation of misfolded transthyretin (TTR) protein, leading to irreversible cardiovascular damage over time.

The Committee for Medicinal Products for Human Use (CHMP) has advised that vutrisiran be authorized for treating both wild type and hereditary forms of ATTR-CM. This therapeutic intervention involves a subcutaneous injection administered quarterly, and it functions by leveraging the body's natural mechanisms to significantly reduce TTR production at its origin.

Vutrisiran is already recognized in the European Union under the brand name Amvuttra for addressing hereditary TTR-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy. Alnylam expresses optimism that vutrisiran could become the first and only RNAi therapeutic to secure European Commission (EC) approval specifically for ATTR-CM.

The CHMP's favorable recommendation was underpinned by data from the phase 3 HELIOS-B trial, which successfully achieved all ten of its pre-specified primary and secondary endpoints. This trial evaluated both the overall population and those receiving the therapy as a standalone treatment. Notably, among the total study demographic, which included patients on Amvuttra monotherapy as well as those on standard treatments, vutrisiran was associated with a 28% reduction in the risk of all-cause mortality and recurrent cardiovascular events during the double-blind treatment period, lasting up to 36 months.

Focusing on patients using the drug as a monotherapy, Amvuttra was shown to lower the risk of all-cause mortality and recurrent cardiovascular events by 33% during the double-blind period and decreased the mortality risk by 35% by month 42. Additionally, in the overall study population, mortality was reduced by 36% at the 42-month mark.

Dr. Pushkal Garg, Alnylam's Chief Medical Officer, highlighted that the HELIOS-B study demonstrated that vutrisiran effectively caused a rapid reduction in TTR levels, enhancing survival rates, decreasing hospital admissions, and slowing disease progression in patients with ATTR-CM. Approximately half of these patients were also on a TTR stabilizer. Dr. Garg emphasized the potential of vutrisiran to serve as a critical treatment option in the EU, given its quarterly dosing schedule and established safety profile.

With the CHMP's recommendation, the European Commission will now review the proposal, moving towards a final decision on the authorization of vutrisiran for this specific use. The potential approval marks a promising advancement in the therapeutic landscape for ATTR-CM, offering hope for better management of this life-threatening condition.