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Amvuttra: how long were the pivotal tox studies?

vutrisiran · TTR (siRNA)
Longest pivotal study
40 weeks
“In the pivotal general toxicology studies of SC vutrisiran in rat (13- and 26-week) and cynomolgus monkey (13- and 40-week), non-adverse findings were observed in the liver (both species), kidneys (rat), and lymph nodes (monkey).”
✓ VerifiedFDA pharmacology/toxicology review, p. 9Source ↗
Pivotal GLP studies
Rat 13-week and 26-week SC monthly; monkey 13-week and 9-month (40-week) SC every 4 weeks
“Frequency of dosing: Every 4 weeks, Day 1 to Day 281 Route of administration: SC injection to the dorsal region”
✓ VerifiedFDA pharmacology/toxicology review, p. 37Source ↗
Route in tox
Subcutaneous, monthly (every 4 weeks)
“Frequency of dosing: Every 4 weeks, Day 1 to Day 281 Route of administration: SC injection to the dorsal region”
✓ VerifiedFDA pharmacology/toxicology review, p. 37Source ↗

Same question, other oligonucleotides

Dawnzera

donidalorsen · Prekallikrein / KLKB1 (ASO)
39 weeks

Givlaari

givosiran · ALAS1 (siRNA)
39 weeks

Izervay

avacincaptad pegol · C5 (aptamer)
39 weeks

Kynamro

mipomersen · APOB (ASO)
52 weeks

Leqvio

inclisiran · PCSK9 (siRNA)
40 weeks

Macugen

pegaptanib · VEGF-165 (aptamer)
39 weeks

Onpattro

patisiran · TTR (siRNA)
39 weeks

Oxlumo

lumasiran · HAO1 (siRNA)
36 weeks

Qalsody

tofersen · SOD1 (ASO)
39 weeks

Qfitlia

fitusiran · Antithrombin / SERPINC1 (siRNA)
39 weeks

Redemplo

plozasiran · APOC3 (siRNA)
37 weeks

Rivfloza

nedosiran · LDHA (siRNA)
39 weeks

Rytelo

imetelstat · Telomerase (oligo)
39 weeks

Spinraza

nusinersen · SMN2 (ASO)
53 weeks

Tegsedi

inotersen · TTR (ASO)
39 weeks

Viltepso

viltolarsen · DMD exon 53 (PMO)
39 weeks

Wainua

eplontersen · TTR (ASO)
39 weeks

Waylivra

volanesorsen · APOC3 (ASO)
39 weeks

Summary across all 24 oligonucleotides →