hATTR-PN patients benefit from long-term use of approved treatment: Data

Long-term treatment with Amvuttra (vutrisiran) was found to be safe and to sustain disease stability in people with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN).

That’s according to up to five years of data from the Phase 3 HELIOS-A clinical trial (NCT03759379), in which patients showed a sustained improvement in nerve function, mobility, and quality of life.

These results “demonstrate relative stability with only modest changes in disease activity, sustained [reductions in the faulty transthyretin, or TTR, protein], and an acceptable safety profile with long-term [Amvuttra] treatment in patients with [hATTR-PN],” the researchers wrote.