Drug Detail:Viltolarsen (Viltolarsen [ vil-toe-lar-sen ])
Drug Class: Miscellaneous uncategorized agents
Usual Adult Dose for Muscular Dystrophy
80 mg/kg via IV infusion once a week
Comments:
- This drug has been approved under an accelerated approval process based on demonstrated increased dystrophin production in skeletal muscle observed in treated patients; continued approval may be contingent upon verification and description of clinical benefit in a confirmatory trial.
Use: For the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.
Usual Pediatric Dose for Muscular Dystrophy
80 mg/kg via IV infusion once a week
Comments:
- This drug has been approved under an accelerated approval process based on demonstrated increased dystrophin production in skeletal muscle observed in treated patients; continued approval may be contingent upon verification and description of clinical benefit in a confirmatory trial.
Use: For the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.
Renal Dose Adjustments
Renal impairment is expected to result in increasing exposure of this drug, however, no specific dosage adjustment can be recommended based on glomerular filtration rate estimated by serum creatinine; frequent monitoring is recommended
Liver Dose Adjustments
No adjustment recommended
Dose Adjustments
Elderly: There is no experience with this drug in elderly patients; Duchenne muscular dystrophy is predominantly a disease of children and young adults
Precautions
CONTRAINDICATIONS: None
Consult WARNINGS section for additional precautions
Dialysis
Data not available
Other Comments
Administration advice:
- Administer once a week as a 60-minute IV infusion
- May be administered using a peripheral or central venous catheter; filtration is not required
- Missed dose: If a dose is missed, administer as soon as possible
Storage requirements:
- Prior to use: Vials should be stored at 2C to 8C (36F to 46F); do not freeze
- Diluted solution may be stored at 20C to 26C (68F to 79F) for up to 6 hours (including infusion time)
- Diluted solution may be stored at 2C to 8C (36F to 46F) for up to 24 hours; do not freeze
Reconstitution/preparation techniques:
- Allow vials to warm to room temperature prior to preparing infusion; mix contents of vial by gently inverting; do not shake
- Volume of infusion should total 100 mL; if volume of drug is less than 100 mL, dilute in 0.9% sodium chloride
- This drug contains no preservatives; infusion should begin as soon as possible
IV compatibility:
- Mix only with 0.9% sodium chloride injection
- Do not mix with other medications or infuse other medications concomitantly
General:
- Drug approval is based on an increase in dystrophin production in skeletal muscle observed in patients treated with this drug; continued approval may be contingent upon verification and description of clinical benefit in a confirmatory trial.
- In clinical trials (n=16), all patients demonstrated an increase in dystrophin levels over their baseline levels.
Monitoring:
- Prior to therapy: Measure serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio; consider measuring glomerular filtration rate using an exogenous filtration marker
- During therapy, obtain urine dipstick monthly; monitor serum cystatin C and urine protein-to-creatinine ratio every 3 months
Patient advice:
- Patients/caregivers should be informed that drugs like this drug have caused renal toxicity and therefore it is important to monitor kidney function closely.
Frequently asked questions
- What are the new drugs for Duchenne muscular dystrophy?
- How long does it take for Viltepso to work?
