Alnylam Pharmaceuticals has secured European Commission (EC) approval for Amvuttra (vutrisiran) to treat transthyretin (ATTR) amyloidosis with cardiomyopathy.
Amvuttra is an RNAi therapeutic that delivers rapid knockdown of variant and wild type transthyretin (TTR), addressing the underlying cause of ATTR amyloidosis.
It follows the European Medicines Agency’s Committee for Orphan Medicinal Products’ positive opinion on maintaining the EU Orphan Designation for Amvuttra.
With the approval, Amvuttra becomes the first RNAi therapeutic approved by the EC specifically for cardiomyopathy manifestations of ATTR amyloidosis.
Alnylam chief medical officer Pushkal Garg said: “Estimates show approximately 100,000 people are affected by ATTR amyloidosis across Europe, most with cardiomyopathy, so this approval marks a critical step toward addressing this underserved patient population.
“Amvuttra is supported by a well-established efficacy and safety profile, with over 6,000 patient-years of global experience in the treatment of hATTR with polyneuropathy.
“By delivering rapid and sustained knockdown of TTR through convenient, quarterly dosing, it offers a clinically differentiated approach with the potential to transform outcomes for patients living with this debilitating and potentially fatal disease.”
The EC approval is based on positive outcomes from the HELIOS-B Phase 3 study, a randomised, double-blind, placebo-controlled, multicentre trial.
The study showed that Amvuttra met all ten pre-specified primary and secondary endpoints, including significant reductions in all-cause mortality and recurrent cardiovascular events.
Also, Amvuttra resulted in improvements in functional capacity, health status, quality of life, and heart failure symptoms.
In the overall population, Amvuttra achieved a 28% reduction in the primary composite of all-cause mortality and recurrent cardiovascular events compared to placebo.
The drug helped reduce the mortality by 36% through 42 months, as per a pre-specified secondary endpoint analysis.
Adverse reactions to Amvuttra include injection site reactions and increased blood alkaline phosphatase and alanine transaminase levels.
HELIOS-B investigator Marianna Fontana said: “The HELIOS-B findings provide compelling evidence to support the use of vutrisiran as a first-line treatment option for patients with ATTR-CM.
“As a physician, it is a privilege to see the true impact on patients in the clinic. The trial enrolled a broad population reflective of real-world clinical practice, and that’s what makes the results so meaningful.
“This is a milestone for patients, who now have a new treatment option that has the potential to significantly improve outcomes of this disease.”
Amvuttra is approved in over 15 countries for treating polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.
It is also approved in the US and Brazil for treating wild-type or hereditary ATTR amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
In the EU, Amvuttra is administered once every three months, either by a healthcare professional or through self-administration by patients or their caregivers.