Drug updated on 12/11/2024
| 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
- This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45 [see Clinical Studies (14)]
- Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials
Latest News
Summary
- This summary is based on the review of one randomized controlled trial. [1]
- Casimersen demonstrated a favorable safety profile in patients aged 7-21 years with Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping. Over 91.4% of treatment-emergent adverse events (TEAEs) were mild and unrelated to the drug, with no serious adverse events or dose reductions reported.
- Plasma pharmacokinetics showed dose-proportional increases in casimersen concentrations, with no evidence of accumulation over the treatment period, supporting its tolerability and consistent drug exposure in this population.
- In the study, 91.4% of TEAEs in both the casimersen and placebo groups were mild and unrelated to the drug, with no casimersen-related serious adverse events (SAEs) or deaths reported.
- No abnormalities in laboratory parameters or vital signs were observed, and no dose reductions were necessary due to adverse events, indicating casimersen was well tolerated in patients aged 7-21 years with Duchenne muscular dystrophy amenable to exon 45 skipping.
- The study included participants aged 7-21 years with DMD amenable to exon 45 skipping, including those with limited ambulation or nonambulatory status. Casimersen was well tolerated in this population, with dose-proportional plasma exposure and no evidence of excessive accumulation over time, suggesting its suitability for long-term use in these patients.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Amondys 45 (casimersen) Prescribing Information. | 2024 | 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