Amvuttra
vutrisiran · TTR (siRNA)40 weeks
Amondys 45: how long were the pivotal tox studies?
casimersen · DMD exon 45 (PMO)
Longest pivotal study
39 weeks
“Title: SRP-4045: A 39-week intravenous infusion toxicity study in male cynomolgus monkeys with an 8-week recovery period”
Pivotal GLP studies
12-wk IV, 22-wk IV (terminated) and 26-wk SC mouse; 12-wk and 39-wk IV monkey; 10-wk IV juvenile rat
“general toxicology (12-, 22-, and 26-week IV in mouse; 12- and 39-week IV in monkey) studies”
Route in tox
IV bolus/30-min infusion; SC for 26-week mouse because chronic IV infeasible (injection-site toxicity)
“Although the clinical route of administration is IV, the adverse effects at the injection site in the 22-week mouse IV study, indicated that the IV route for the 26-week duration is not feasible.”
Same question, other oligonucleotides
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Tegsedi
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Wainua
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Waylivra
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Zanvastro
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