Kynamro
mipomersen · APOB (ASO)Applicant estimated safe starting dose from nonclinical results; method not specified
Dawnzera: how was the first human dose set?
donidalorsen · Prekallikrein / KLKB1 (ASO)
First-in-human starting dose
Not stated in public reviews
NOAEL / key findings
Monkey NOAEL 6 mg/kg/week (platelet reductions at higher doses; ~29-fold margin); mouse NOAEL top dose; liver and kidney target organs
“The NOAEL for donidalorsen was determined to be 6 mg/kg/week in monkeys, based on PLT reductions”
Same question, other oligonucleotides
Zanvastro
zilganersen · GFAP (ASO)Starting-dose rationale not stated; FDA capped clinical IT dose at 25 mg (~1/10 mouse/monkey NOAEL), later 50 mg Q12W
Amondys 45
casimersen · DMD exon 45 (PMO)Not stated in public reviews
Amvuttra
vutrisiran · TTR (siRNA)Not stated in public reviews
Exondys 51
eteplirsen · DMD exon 51 (PMO)Not stated in public reviews
Givlaari
givosiran · ALAS1 (siRNA)Not stated in public reviews
Izervay
avacincaptad pegol · C5 (aptamer)Not stated in public reviews
Leqvio
inclisiran · PCSK9 (siRNA)Not stated in public reviews
Macugen
pegaptanib · VEGF-165 (aptamer)Not stated in public reviews
Onpattro
patisiran · TTR (siRNA)Not stated in public reviews
Oxlumo
lumasiran · HAO1 (siRNA)Not stated in public reviews
Qalsody
tofersen · SOD1 (ASO)Not stated in public reviews
Qfitlia
fitusiran · Antithrombin / SERPINC1 (siRNA)Not stated in public reviews
Redemplo
plozasiran · APOC3 (siRNA)Not stated in public reviews
Rivfloza
nedosiran · LDHA (siRNA)Not stated in public reviews
Rytelo
imetelstat · Telomerase (oligo)Not stated in public reviews
Spinraza
nusinersen · SMN2 (ASO)Not stated in public reviews
Tegsedi
inotersen · TTR (ASO)Not stated in public reviews
Tryngolza
olezarsen · APOC3 (ASO)Not stated in public reviews
Viltepso
viltolarsen · DMD exon 53 (PMO)Not stated in public reviews
Vyondys 53
golodirsen · DMD exon 53 (PMO)Not stated in public reviews
Wainua
eplontersen · TTR (ASO)Not stated in public reviews
Waylivra
volanesorsen · APOC3 (ASO)Not stated in public reviews