Amondys 45
casimersen · DMD exon 45 (PMO)39 weeks
Izervay: how long were the pivotal tox studies?
avacincaptad pegol · C5 (aptamer)
Longest pivotal study
39 weeks
“Study title: A 1-Month (Single-Dose) and 9-Month (Ten-Dose) Toxicity Study of ARC1905 Given by Intravitreal Injections to Rabbits”
Pivotal GLP studies
GLP 1-month and 9-month (10 monthly doses) IVT studies in rabbit and dog +/- Lucentis; 7-day IV rat and monkey studies
“Repeat-dose IVT toxicity studies were conducted in New Zealand White rabbits (0, 0.15, 0.5, and 1.5 mg/eye) and Beagle dogs (0, 0.3, 1, and 3.0 mg/eye).”
Route in tox
Intravitreal (ocular studies); IV bolus plus continuous infusion (systemic studies)
“Repeat-dose IVT toxicity studies were conducted in New Zealand White rabbits (0, 0.15, 0.5, and 1.5 mg/eye) and Beagle dogs (0, 0.3, 1, and 3.0 mg/eye).”
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
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
Waylivra
volanesorsen · APOC3 (ASO)39 weeks
Zanvastro
zilganersen · GFAP (ASO)39 weeks