Amondys 45
casimersen · DMD exon 45 (PMO)39 weeks
Qfitlia: how long were the pivotal tox studies?
fitusiran · Antithrombin / SERPINC1 (siRNA)
Longest pivotal study
39 weeks
“Repeat-dose toxicology studies were conducted in healthy rats for up to 26 weeks, healthy monkeys for up to 39 weeks, and in hemophilia mice for up to 26 weeks.”
Pivotal GLP studies
GLP SC weekly: rat 26-week with FEED (13-wk recovery), monkey 39-week (13-wk recovery), 7-week rat and monkey studies, 26-week hemophilia A mouse
“A 26-Week Subcutaneous Injection Toxicity, Toxicokinetic, and Fertility and Early Embryonic Development (to Implantation) Study in the Rat Followed By a 13-Week Recovery Period”
Route in tox
SC (weekly dosing)
“Animals were dosed once weekly by the SC route.”
Same question, other oligonucleotides
Amvuttra
vutrisiran · TTR (siRNA)40 weeks
Dawnzera
donidalorsen · Prekallikrein / KLKB1 (ASO)39 weeks
Exondys 51
eteplirsen · DMD exon 51 (PMO)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
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