Amondys 45
casimersen · DMD exon 45 (PMO)39 weeks
Redemplo: how long were the pivotal tox studies?
plozasiran · APOC3 (siRNA)
Longest pivotal study
37 weeks
“Q4W in rats (15, 30, and 120 mg/kg) and monkeys (30, 90, and 180 mg/kg) for durations up to 24- and 37-weeks, respectively”
Pivotal GLP studies
24-week rat and 37-week monkey SC Q4W repeat-dose toxicity studies
“Repeat-dose general toxicology studies with plozasiran were conducted with SC administration (consistent with the clinical route) Q4W in rats”
Route in tox
Subcutaneous, Q4W
“Repeat-dose general toxicology studies with plozasiran were conducted with SC administration (consistent with the clinical route) Q4W in rats”
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
Qfitlia
fitusiran · Antithrombin / SERPINC1 (siRNA)39 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