EU approves Amvuttra for hereditary transthyretin-mediated (hATTR) amyloidosis

The EC approval is based on positive 18-month results from the HELIOS-A Phase III study, where Amvuttra significantly improved the signs and symptoms of hATTR amyloidosis, with more than 50 percent of patients experiencing halting or reversal of their polyneuropathy manifestations.

In the HELIOS-A study, Amvuttra met the primary and all secondary endpoints of the study at both 9 months and 18 months, demonstrating reversal in neuropathy impairment and an encouraging safety and tolerability profile. Amvuttra demonstrated improvement in the mean change from baseline in modified Neuropathy Impairment Score + 7 (mNIS+7) at 18 months (the primary endpoint for the EU), as compared to external placebo data from the landmark APOLLO Phase III study of patisiran.

After 18 months of dosing, the most frequently occurring adverse reactions in Amvuttra-treated patients were arthralgia (joint stiffness) and pain in extremity (pain in arms and legs). Other less frequent adverse reactions reported with Amvuttra were dyspnea (shortness of breath), injection site reaction and an increase in blood alkaline phosphatase (a liver enzyme).