Alnylam Pharmaceuticals has received marketing authorisation from the European Commission (EC) for RNAi therapeutic, Amvuttra (vutrisiran), to treat hereditary transthyretin-mediated (hATTR) amyloidosis.
Amvuttra targets and supresses specific messenger RNA (mRNA) that blocks the wild-type and variant transthyretin (TTR) protein production before it is made.
It uses the company’s Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform.
The RNAi therapeutic is indicated to treat hATTR amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy.
The marketing approval follows the Committee for Medicinal Products for Human Use (CHMP) of European Medicines Agency’s (EMA) positive opinion on Amvuttra, in July this year.
Alnylam International Region head and senior vice-president Kasha Witkos said: “We are committed to delivering continued innovation to people affected by rare, life-limiting conditions where high unmet need still exists.
“Today’s decision now means we can progress working with health authorities across Europe to achieve responsible and sustainable access arrangements that allow us to bring Amvuttra to patients as quickly as possible.”
The regulatory approval is based on positive 18-month data obtained from the HELIOS-A Phase III study.
The randomised, multicentre, open-label, international HELIOS-A study was designed to assess Amvuttra’s efficacy and safety across a group of hATTR amyloidosis patients with stage 1 or stage 2 polyneuropathy.
In the trial, Amvuttra significantly improved hATTR amyloidosis’ symptoms and signs.
Over 50% of the participants in the trial have experienced halting or reversal of their polyneuropathy manifestations.
The RNAi therapeutic has also met the trial’s primary and all secondary endpoints at both nine months and 18 months.
It showed reversal in neuropathy impairment as well as an encouraging safety and tolerability profile.
In June, the US Food and Drug Administration (FDA) approved Amvuttra to treat polyneuropathy of hATTR amyloidosis in adult patients.