Global, randomized, open-label, multicenter, Phase 3 study1,2
aStudy patients were ≥18 years of age, had a diagnosis of hATTR amyloidosis with polyneuropathy caused by any TTR variant, a Polyneuropathy Disability (PND) score ≤IIIb, a Neuropathy Impairment Score (NIS) of 5–130, a Karnofsky Performance Status (KPS) score ≥60%, and were permitted to have previously used TTR stabilizers.3
BMI=body mass index; IV=intravenous; q3m=every 3 months; q3w=every 3 weeks; SC=subcutaneous.
aStudy patients were ≥18 years of age, had a diagnosis of hATTR amyloidosis with polyneuropathy caused by any TTR variant, a Polyneuropathy Disability (PND) score ≤IIIb, a Neuropathy Impairment Score (NIS) of 5–130, a Karnofsky Performance Status (KPS) score ≥60%, and were permitted to have previously used TTR stabilizers.3
BMI=body mass index; IV=intravenous; q3m=every 3 months; q3w=every 3 weeks; SC=subcutaneous.
- 164 patients with the polyneuropathy of hATTR amyloidosis were randomized 3:1 to the AMVUTTRA group or the patisiran reference group1
- Efficacy assessments were based on a comparison of the AMVUTTRA arm of the study with an external placebo group from APOLLO (n=77), a randomized controlled study in a comparable patient population1,a
- The patisiran reference group was included in the study to validate the use of the external placebo group2
- 96% of AMVUTTRA-treated patients completed at least 18 months of treatment2
Participants in the study were representative of the real-world population of patients with the polyneuropathy of hATTR amyloidosis.1,2
aStudy patients were ≥18 years of age, had a diagnosis of hATTR amyloidosis with polyneuropathy caused by any TTR variant, a Polyneuropathy Disability (PND) score ≤IIIb, a Neuropathy Impairment Score (NIS) of 5–130, a Karnofsky Performance Status (KPS) score ≥60%, and were permitted to have previously used TTR stabilizers.3
BMI=body mass index; IV=intravenous; q3m=every 3 months; q3w=every 3 weeks; SC=subcutaneous.