Alnylam Reports Results of Vutrisiran in P-III HELIOS-A Study for the Treatment of hATTR Amyloidosis with Polyneuropathy

Shots:

  • The P-III HELIOS-A study evaluate vutrisiran (25mg, SC, q3mos.) vs patisiran (0.3 mg/kg, IV, q3w) in a ratio (3:1) in 164 patients with hATTR amyloidosis with polyneuropathy at 57 sites in 22 countries
  • The trial met all 2EPs i.e., @ 18mos., therapy showed improvements in neuropathy as measured by mNIS+7, QoL, gait speed, nutritional status & overall disability compared to PBO; reduction in serum TTR levels & showed non-inferiority over  patisiran
  • The therapy showed improvements in exploratory EPs @18mos., including biomarker NT-proBNP, echocardiographic parameters & technetium uptake in a planned cohort. The therapy is currently under review by multiple regulatory authorities globally

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