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. 2022 Jun;50(6):888–897. doi: 10.1124/dmd.121.000417

TABLE 1.

Local and systematic absorption of the FDA-approved ASO drugs

The data are extracted from the FDA drug labels of each ASO drug.

Drug AdministrationSite and Route Local vs. Systematic Absorption Ability to Cross Blood-Brain Barrier Tissues with Highest Drug Concentrations Peak Plasma Concentration Time (Hours)
Fomivirsen Intravitreal injection Local eye No Retina, iris; systemic exposure: minimal (below limits of quantitation) Very low
Pegaptanib Intravitreal injection Local eye No Vitreous fluid, retina, aqueous fluid; systemic exposure: kidney Slowly absorbed into systemic circulation
Nusinersen Intrathecal injection CNS and systematic Yes CNS, plasma, peripheral tissue (skeletal muscle, liver, kidney), fat, bone marrow, spleen 1.7–6
Mipomersen S.C. injection Systematic No Liver, kidney, bone marrow, adipose tissue, lymph nodes 3–4
Inotersen S.C. injection Systematic No Liver, kidney, broad tissues 2–4
Defibrotide I.V. infusion Systematic No Cells lining blood vessel in liver 2
Eteplirsen I.V. infusion Systematic No Kidney, skeletal muscles 1–2
Golodirsen I.V. infusion Systematic No Kidney, all tissue except CNS 1
Viltolarsen I.V. infusion Systematic No Kidney, skeletal muscle 1
Casimersen I.V. infusion Systematic No Kidney, skeletal muscle 1