Vutrisiran is an anti-transthyretin small interfering ribonucleic acid (siRNA) agent. It is indicated for polyneuropathy caused by hereditary transthyretin-related amyloidosis (hATTR) in adults. Amyloidogenic TTR mutations cause deposits primarily in the peripheral nerves. siRNA agents cause degradation of mutant and wild-type TTR mRNA through RNA interference.
Approval was based on results from HELIOS-A, a global, randomized, open-label, multicenter, phase 3 study. A total of 164 patients with hATTR amyloidosis were randomized 3:1 to receive either vutrisiran 25 mg SC every 3 months (n = 122) or patisiran 0.3 mg/kg IV every 3 weeks (n = 42) for 18 months. The efficacy of vutrisiran was also assessed by comparing the vutrisiran group in HELIOS-A with the placebo group (n = 77) from the APOLLO phase 3 study of patisiran.
Vutrisiran met the primary endpoint of the study, the change from baseline in the modified Neuropathy Impairment Score + 7 (mNIS+7) at 9 months. Treatment with vutrisiran (n = 114) resulted in a 2.2 point mean decrease (improvement) in mNIS+7 from baseline, compared to a 14.8 point mean increase (worsening) reported for the external placebo group (n = 67), resulting in a 17.0 point mean difference relative to placebo (P<0.0001). By 9 months, 50% of patients treated with vutrisiran experienced improvement in neuropathy impairment relative to baseline. Neurology 2021 Apr 13;96(15 suppl)
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Cite this: FDA Drug Approvals, Rheumatology — 2022 Midyear Review - Medscape - Aug 24, 2022.