Amondys 45
casimersen · DMD exon 45 (PMO)39 weeks
Leqvio: how long were the pivotal tox studies?
inclisiran · PCSK9 (siRNA)
Longest pivotal study
40 weeks
“Inclisiran was also well-tolerated in repeat dose studies up to 29-weeks of duration in rats and 40-weeks of duration in monkeys.”
Pivotal GLP studies
Rat 29-week and monkey 40-week SC Q4W (13-week recovery); plus 4- and 15-week studies and 13-week monkey statin combination
“Toxicological characterization of inclisiran was performed in studies of 29 weeks in rats and 40 weeks in NHPs at doses up to 8 times (≤ 250 mg/kg Q4W) and 19 times (300 mg/kg Q4W) the MRHD (based on BSA), respectively.”
Route in tox
Subcutaneous, weekly to every 4 weeks (more frequent than clinical Q6M)
“It is important to note that animals were dosed more frequently in toxicology studies (QD to Q4W) than in clinical studies (Q3M or Q6M).”
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
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
Waylivra
volanesorsen · APOC3 (ASO)39 weeks
Zanvastro
zilganersen · GFAP (ASO)39 weeks