Drug updated on 3/28/2024
Dosage Form | Injection (intravenous; 100 mg/2 mL) |
Drug Class | Antisense oligonucleotides |
Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated 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.
Summary
- Casimersen (Amondys 45) is designed to bypass frameshift DMD mutations and produce internally truncated, yet functional, dystrophin protein in patients amenable to exon 45 skipping.
- One Randomized Controlled Trial document was reviewed for this analysis which focused on the safety, tolerability and plasma pharmacokinetics of casimersen (Amondys 45).
- The trial enrolled a total of 12 participants aged between 7-21 years who had limited ambulation or were nonambulatory due to Duchenne muscular dystrophy (DMD).
- During the double-blind dose titration phase of the study, participants were randomized into two groups with one receiving weekly casimersen infusions at escalating doses while the other received placebo.
- Treatment-emergent adverse events occurred in all participants but they were mostly mild and unrelated to casimersen; there were no deaths or serious AEs related to its use indicating that it was well tolerated by patients with DMD amenable to exon 45 skipping.
- Plasma exposure from casimersen increased proportionally with dosage without any suggestion of accumulation over time suggesting consistent pharmacokinetic properties across different dosages used during treatment periods up till week sixty.
Product Monograph / Prescribing Information
Document Title | Year | Source |
---|---|---|
Amondys 45 (casimersen) Prescribing Information. | 2023 | Sarepta Therapeutics, Inc. Cambridge, MA |
Randomized Controlled Trials
Document Title | Sex Distribution | Year | Source |
---|---|---|---|
Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: a randomized, double‐blind, placebo‐controlled, dose‐titration trial. | 12Subjects F: 0% M: 100% | 2021 | Muscle & Nerve |
Sex Distribution:
F:0%
M:100%
12Subjects
Year:
2021
Source:Muscle & Nerve