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Oxlumo: how long were the pivotal tox studies?

lumasiran · HAO1 (siRNA)
Longest pivotal study
36 weeks
“Chronic administration of lumasiran was assessed in good laboratory practice (GLP) repeat-dose toxicology studies up to 25- and 36-weeks in rats and monkeys, respectively.”
✓ VerifiedFDA integrated review, p. 56Source ↗
Pivotal GLP studies
Rat 25-week SC monthly; monkey 36-week SC every 4 weeks (10 doses); plus 8-week weekly monkey study
“Dose and frequency of dosing: 30, 100, or 300 mg/kg lumasiran once every 4 weeks for a total of 10 doses Route of administration: SC”
✓ VerifiedFDA integrated review, p. 86Source ↗
Route in tox
Subcutaneous, weekly or monthly, matching clinical route
“Dose and frequency of dosing: 0, 20, 50 and 200 mg/kg Once/month for 25 weeks Route of administration: SC”
✓ VerifiedFDA integrated review, p. 83Source ↗

Same question, other oligonucleotides

Amvuttra

vutrisiran · TTR (siRNA)
40 weeks

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

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 →