Positive Phase III results for vutrisiran in the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy

In the HELIOS-A study, vutrisiran met the primary endpoint (change in modified Neuropathy Impairment Score at 9 months versus historical placebo data) and all secondary endpoints. The company intends to submit a New Drug Application to the US FDA on the basis of these findings.

Source:

Biospace Inc.