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Longest pivotal study
39 weeks
“In a 39-week monkey toxicity study that included a 26-week recovery period, olezarsen induced a marked reduction in platelet count (>98% decrease) in a single male animal at the 12 mg/kg/wk”
✓ VerifiedFDA integrated review, p. 102Source ↗
Pivotal GLP studies
GLP weekly SC: CD-1 mouse 26 weeks (13-wk recovery); cynomolgus monkey 39 weeks (26-wk recovery)
“The nonclinical evaluation of the safety of olezarsen included repeat-dose general toxicology studies of up to 26 and 39-weeks in duration with 13 and 26-week recovery periods in mice and monkeys, respectively”
✓ VerifiedFDA integrated review, p. 102Source ↗
Route in tox
Subcutaneous (once weekly)
“once-weekly SC injections. These studies included 13- and 26-week recovery periods in the mouse and monkey, respectively.”
✓ VerifiedFDA integrated review, p. 220Source ↗

Same question, other oligonucleotides

Amvuttra

vutrisiran · TTR (siRNA)
40 weeks

Dawnzera

donidalorsen · Prekallikrein / KLKB1 (ASO)
39 weeks

Defitelio

defibrotide · Fragmented DNA oligonucleotides
13 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

Vitravene

fomivirsen · CMV IE2 mRNA (ASO)
13 weeks

Wainua

eplontersen · TTR (ASO)
39 weeks

Waylivra

volanesorsen · APOC3 (ASO)
39 weeks

Hibsago

bepirovirsen · HBV RNA (ASO)
Not stated in public reviews

Summary across all 27 oligonucleotides →