Amondys 45
casimersen · DMD exon 45 (PMO)39 weeks
Oxlumo: how long were the pivotal tox studies?
lumasiran · HAO1 (siRNA)
Longest pivotal study
36 weeks
“Chronic administration of lumasiran was assessed in good laboratory practice (GLP) repeat-dose toxicology studies up to 25- and 36-weeks in rats and monkeys, respectively.”
Pivotal GLP studies
Rat 25-week SC monthly; monkey 36-week SC every 4 weeks (10 doses); plus 8-week weekly monkey study
“Dose and frequency of dosing: 30, 100, or 300 mg/kg lumasiran once every 4 weeks for a total of 10 doses Route of administration: SC”
Route in tox
Subcutaneous, weekly or monthly, matching clinical route
“Dose and frequency of dosing: 0, 20, 50 and 200 mg/kg Once/month for 25 weeks Route of administration: SC”
Same question, other oligonucleotides
Amvuttra
vutrisiran · TTR (siRNA)40 weeks
Dawnzera
donidalorsen · Prekallikrein / KLKB1 (ASO)39 weeks
Defitelio
defibrotide · Fragmented DNA oligonucleotides13 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
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
Vitravene
fomivirsen · CMV IE2 mRNA (ASO)13 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
Hibsago
bepirovirsen · HBV RNA (ASO)Not stated in public reviews