Alnylam Pharmaceuticals’ RNAi therapeutic vutrisiran has been recommended by the European Medicines Agency’s human medicines committee to treat adults with transthyretin amyloid cardiomyopathy (ATTR-CM).
ATTR-CM is a potentially fatal disease of the heart muscle that occurs when misfolded transthyretin (TTR) protein accumulates and causes irreversible cardiovascular damage over time.
The Committee for Medicinal Products for Human Use (CHMP) has recommended that the drug be authorised to treat both wild type and hereditary ATTR-CM.
Administered as a subcutaneous injection once every three months, vutrisiran is designed to work with the body’s natural system to knock down TTR at its source.
The drug is already approved in the EU under the brand name Amvuttra to treat hereditary TTR-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, and “has the potential” to be the first and only RNAi therapeutic to receive European Commission (EC) approval for ATTR-CM, according to Alnylam.
The CHMP’s decision in ATTR-CM was supported by results from the phase 3 HELIOS-B trial, which met all ten of its pre-specified primary and secondary endpoints across both the overall and monotherapy populations.
In the overall study population, which included patients receiving Amvuttra as a monotherapy and those using the therapy alongside standard treatments, Alnylam’s drug reduced the risk of all-cause mortality (ACM) and recurrent cardiovascular events by 28% during the double-blind treatment period of up to 36 months.
In the monotherapy population, Amvuttra reduced the risk of ACM and recurrent cardiovascular events by 33% in the double-blind period and the risk of mortality by 35% at month 42. Mortality in the overall population was also reduced by 36% at month 42.
Alnylam’s chief medical officer, Pushkal Garg, said: “In the HELIOS-B study, vutrisiran treatment resulted in rapid knockdown of TTR and led to improved survival, fewer hospitalisations and less disease progression in patients with ATTR-CM, nearly half of whom were on a TTR stabiliser.
“Combined with its quarterly dosing and well-established safety profile, we believe vutrisiran could offer an important new treatment option for patients in the EU.”
The EC will now review the CHMP’s recommendation as it makes a final decision on vutrisiran in this indication.
