Usual Adult Dose for Muscular Dystrophy

30 mg/kg via IV infusion once a week

Comments:

• This drug has been approved under an accelerated approval based on an increase in dystrophin production in skeletal muscle; continued approval may be contingent upon verification of a clinical benefit in confirmatory trials.

Use: For the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping.

Usual Pediatric Dose for Muscular Dystrophy

30 mg/kg via IV infusion once a week

Comments:

• This drug has been approved under an accelerated approval based on an increase in dystrophin production in skeletal muscle; continued approval may be contingent upon verification of a clinical benefit in confirmatory trials.

Use: For the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping.

Renal Dose Adjustments

Patients with known renal impairment should be closely monitored during treatment; no specific dosage adjustments can be made based on estimated glomerular filtration rate

Liver Dose Adjustments

No adjustment recommended

Precautions

CONTRAINDICATIONS: None

Consult WARNINGS section for additional precautions.

Dialysis

Data not available

Other Comments

Administration advice:

• Administer once weekly as an IV infusion over 35 to 60 minutes
• A topical anesthetic cream may be applied to the infusion site prior to administration
• Flush IV line with 0.9% Sodium Chloride Injection before and after infusion; an in-line 0.2-micron filter should be used

Missed Dose: If a dose is missed administer as soon as possible after the scheduled dose

Storage requirements:
Unopened vials: Store at 36F to 46F (2C to 8C) in original carton; protect from light; do not freeze
Diluted Solution: May store for up to 24 hours at 36F to 46F (2C to 8C); do not freeze

Reconstitution/preparation techniques:

• Must be diluted prior to administration
• Allow vials to warm to room temperature; gently invert vials to mix contents; do not shake
• Dilute the required volume of drug with 0.9% sodium chloride injection to make a total volume of 100 to 150 mL; gently invert to mix; do not shake
• Infuse within 4 hours of dilution; may store refrigerated for up to 24 hours

IV compatibility: Do not mix with other medications or infuse other medications concomitantly via same IV access

General:

• The FDA has granted accelerated approval of this drug based on surrogate endpoint data showing an increase in dystrophin production that is reasonably likely to predict clinical benefit in patients with DMD who have a confirmed mutation of the dystrophin gene amenable to exon 45 skipping.
• Trials are ongoing to verify whether this drug improves motor function in these patients.

Monitoring:

• Prior to therapy, measure serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio (UPCR); consider measuring glomerular filtration rate using an exogenous filtration marker
• Monitor for proteinuria with monthly dipstick urinalysis; if pyrogallol red is the reagent in the test, be sure to use urine obtained at least 48 hours after the most recent infusion; alternatively, use a laboratory test without this reagent to avoid receiving a false positive for urine protein
• Monitor serum cystatin C and UPCR every 3 months

Patient advice:

• Patients/caregivers should understand that nephrotoxicity has occurred with drugs similar to this drug and therefore, it will be important to monitor for kidney toxicity during treatment.

Frequently asked questions

• What are the new drugs for Duchenne muscular dystrophy?