Amondys 45
casimersen · DMD exon 45 (PMO)39 weeks
Waylivra: how long were the pivotal tox studies?
volanesorsen · APOC3 (ASO)
Longest pivotal study
39 weeks
“Cynomolgus monkey: 7 or 9/s/g 0-3-6-12-20 39 wk 3”
Pivotal GLP studies
GLP SC repeat-dose: monkey 13- and 39-week, rat 26-week, mouse 13- and 26-week studies; 2-year mouse and rat carcinogenicity
“Volanesorsen plasma PK following SC administration in male and female monkeys were further studied in the 39-week toxicity study (study 304801-AS11).”
Route in tox
SC (IV loading doses in the 13-week monkey study)
“0-4-8-12-40 (loading IV, subsequent SC, SC only @40)”
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
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
Zanvastro
zilganersen · GFAP (ASO)39 weeks