.Pharmacolibrary.Drugs.S_SensoryOrgans.S01X_OtherOphthalmologicals.S01XA29_Sepofarsen.Sepofarsen .Pharmacolibrary.Drugs.S_SensoryOrgans.S01X_OtherOphthalmologicals.S01XA29_Sepofarsen.Sepofarsen| name: | Sepofarsen |
| ATC code: | S01XA29 | route: | intravitreal |
| n-compartments | 1 |
Sepofarsen is an antisense oligonucleotide designed to target and restore c.2991+1655A>G splicing defect in the CEP290 gene. It is investigated for the treatment of Leber congenital amaurosis type 10 (LCA10), a rare genetic retinal degenerative disorder. Sepofarsen is not currently approved by regulatory authorities for general use.
No human or animal pharmacokinetic parameter data are available in publications for sepofarsen, including standard references, clinicaltrials reporting, and regulatory documents as of mid-2024. Sepofarsen is administered as an intravitreal injection to the eye, where absorption and distribution are mainly local. Systemic exposure is typically negligible for intravitreal oligonucleotide drugs.