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Study length
Oxlumo: how long were the pivotal tox studies?
lumasiran · HAO1 (siRNA)
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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.”
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FDA integrated review, p. 56
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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”
✓ Verified
FDA integrated review, p. 86
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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”
✓ Verified
FDA integrated review, p. 83
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Same question, other oligonucleotides
Amondys 45
casimersen · DMD exon 45 (PMO)
39 weeks
Amvuttra
vutrisiran · TTR (siRNA)
40 weeks
Dawnzera
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39 weeks
Exondys 51
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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
Tryngolza
olezarsen · APOC3 (ASO)
39 weeks
Viltepso
viltolarsen · DMD exon 53 (PMO)
39 weeks
Vyondys 53
golodirsen · DMD exon 53 (PMO)
39 weeks
Wainua
eplontersen · TTR (ASO)
39 weeks
Waylivra
volanesorsen · APOC3 (ASO)
39 weeks
Zanvastro
zilganersen · GFAP (ASO)
39 weeks
Summary across all 24 oligonucleotides →
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