"Alnylam Pharmaceuticals’ Amvuttra (vutrisiran) has been approved by the European Commission (EC) to treat adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a potentially fatal disease of the heart muscle. The RNAi therapeutic has been authorised to treat both wild type and hereditary ATTR-CM, which occurs when misfolded transthyretin (TTR) protein accumulates and causes irreversible cardiovascular damage over time.
• Hereditary ATTR affects approximately 50,000 people worldwide, while wild-type ATTR impacts up to 300,000.
• Patients may present with cardiomyopathy, polyneuropathy or both manifestations of the disease.
Given as a subcutaneous injection once every three months, Amvuttra is designed to work with the body’s natural system to knock down TTR at its source, and is already approved in the EU to treat hereditary TTR-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy.
Alnylam’s chief medical officer, Pushkal Garg, said: “By delivering rapid and sustained knockdown of TTR through convenient, quarterly dosing, [Amvuttra] offers a clinically differentiated approach with the potential to transform outcomes for patients living with this debilitating and potentially fatal disease.
“We now look forward to securing access to Amvuttra for eligible patients across the EU as quickly as possible.”
The EC’s latest decision on the drug follows a recent recommendation from the European Medicines Agency’s human medicines committee and was based on results from the late-stage HELIOS-B trial.
• In the overall study population, which included patients receiving Amvuttra as a monotherapy and those using the therapy alongside standard-of-care treatments such as tafamidis and SGLT2 inhibitors, Alnylam’s drug reduced the risk of all-cause mortality and recurrent cardiovascular events by 28% compared to placebo.
• Mortaliy in this population was also reduced by 36% at month 42 in a pre-specified secondary endpoint analysis, which included up to 36 months of the double-blind period plus six months of open-label extension.
• “The trial enrolled a broad population reflective of real-world clinical practice, and that’s what makes the results so meaningful,” said HELIOS-B investigator, Marianna Fontana.
• “This is a milestone for patients, who now have a new treatment option that has the potential to significantly improve outcomes of this disease.”