Amondys 45

  • Miscellaneous musculoskeletal disorders

Amondys 45 Generic Name & Formulations

General Description

Casimersen 50mg/mL; soln for IV infusion after dilution; preservative-free.

Pharmacological Class

Antisense oligonucleotide.

How Supplied

Single-dose vial (2mL)—1


Generic Availability


Mechanism of Action

Casimersen is designed to bind to exon 45 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 45 skipping.

Amondys 45 Indications


Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping.

Amondys 45 Dosage and Administration

Adults and Children

Give as IV infusion over 35–60mins. 30mg/kg once weekly.


Use via an in-line 0.2 micron filter.

Amondys 45 Contraindications

Not Applicable

Amondys 45 Boxed Warnings

Not Applicable

Amondys 45 Warnings/Precautions


Consider slowing the infusion, interrupting, or discontinuing therapy if hypersensitivity reactions occur; treat appropriately and monitor until resolves. Monitor renal function. Measure serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio, and consider measuring GFR prior to initiation. During treatment, monitor urine dipstick monthly, and serum cystatin C and urine protein-to-creatinine ratio every 3 months. To test urine protein: may use urine samples obtained prior to infusion or at least 48hrs after the most recent infusion. Refer to pediatric nephrologist if a persistent increase in serum cystatin C or proteinuria is detected. Renal impairment: monitor closely. Pregnancy. Nursing mothers.

Amondys 45 Pharmacokinetics


Cmax was reached at the end of infusion following a single IV dose of casimersen. 

Casimersen exposure increased in a proportional manner with dose increment. 


Binding to human plasma protein was not concentration-dependent and ranged from 8.4% to 31.6%.

Mean apparent volume of distribution at steady state: 367 mL/kg (%CV = 28.9).


Casimersen is metabolically stable in human hepatic microsomal incubations.


Renal (>90%). 

Half-life: 3.5 hours (SD 0.4 hours).

Amondys 45 Interactions


Do not mix or infuse with other drugs concomitantly via same IV access line. May cause false (+) results for urine protein with using pyrogallol red reagent.

Amondys 45 Adverse Reactions

Adverse Reactions

Upper RTI, cough, pyrexia, headache, arthralgia, oropharyngeal pain; hypersensitivity reactions, renal toxicity.

Amondys 45 Clinical Trials

See Literature

Amondys 45 Note

Not Applicable

Amondys 45 Patient Counseling

See Literature