.Pharmacolibrary.Drugs.M_MusculoSkeletalSystem.M09A_OtherDrugsForDisordersOfTheMusculoSkeletalSystem.M09AX06_Eteplirsen.Eteplirsen .Pharmacolibrary.Drugs.M_MusculoSkeletalSystem.M09A_OtherDrugsForDisordersOfTheMusculoSkeletalSystem.M09AX06_Eteplirsen.Eteplirsen| name: | Eteplirsen |
| ATC code: | M09AX06 | route: | intravenous |
| n-compartments | 2 |
Eteplirsen is an antisense oligonucleotide designed to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene amenable to exon 51 skipping. It is administered intravenously and is approved for use in several countries including the United States.
Pharmacokinetic parameters derived from clinical studies in pediatric male subjects with Duchenne muscular dystrophy (DMD) aged 7-13 years receiving repeated intravenous infusions.
Patel, Y, et al., & East, L (2025). A Population Pharmacokinetic Model to Inform Extension of the Eteplirsen Dosing Regimen Across the Broad DMD Population. CPT: pharmacometrics & systems pharmacology 14(5) 891–903. DOI:10.1002/psp4.70001 PUBMED:https://pubmed.ncbi.nlm.nih.gov/40009522
Goey, AKL, et al., & East, L (2024). Characterization of Nonclinical Drug Metabolism and Pharmacokinetic Properties of Phosphorodiamidate Morpholino Oligonucleotides, a Novel Drug Class for Duchenne Muscular Dystrophy. Drug metabolism and disposition: the biological fate of chemicals 52(12) 1396–1406. DOI:10.1124/dmd.124.001819 PUBMED:https://pubmed.ncbi.nlm.nih.gov/39516029