name: | Casimersen |
ATC code: | M09AX13 | route: | intravenous |
n-compartments | 1 |
Casimersen is an antisense oligonucleotide used for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. It is approved by the FDA and marketed under the brand name Amondys 45.
Pharmacokinetic parameters estimated from FDA label and public summaries for intravenous administration in male pediatric patients (ages 7–20 years) with Duchenne muscular dystrophy.
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