Version May 2024
Duchenne muscular dystrophy (DMD): Male children ≥7 years and Adolescents: IV: 30 mg/kg/dose once weekly; in the clinical trials, patients ranged in age from 7 to 13 years (Ref).
There are no dosage adjustments provided in the manufacturer's labeling (has not been studied).
There are no dosage adjustments provided in the manufacturer's labeling (has not been studied).
(For additional information see "Eteplirsen: Drug information")
Duchenne muscular dystrophy: IV: 30 mg/kg once weekly.
There are no dosage adjustments provided in the manufacturer’s labeling (has not been studied).
There are no dosage adjustments provided in the manufacturer’s labeling (has not been studied).
The following adverse drug reactions and incidences are derived from product labeling unless otherwise specified. Incidences include reactions experienced with approved and unapproved doses.
>10%:
Dermatologic: Contact dermatitis
Gastrointestinal: Vomiting
Nervous system: Balance impairment
Frequency not defined: Hypersensitivity: Hypersensitivity reaction
Postmarketing:
Cardiovascular: Flushing
Endocrine & metabolic: Dehydration
Genitourinary: Proteinuria
Nervous system: Malaise
Respiratory: Bronchospasm, cyanosis of the lips
Miscellaneous: Fever
There are no contraindications listed in the manufacturer's labeling.
Concerns related to adverse effects:
• Hypersensitivity: Hypersensitivity reactions (including bronchospasm, chest pain, cough, tachycardia, and urticaria) have been reported with use; manage appropriately and consider slowing the infusion rate or interrupting therapy.
Excipient information presented when available (limited, particularly for generics); consult specific product labeling.
Solution, Intravenous [preservative free]:
Exondys 51: 50 mg/mL (2 mL, 10 mL)
No
Solution (Exondys 51 Intravenous)
100 mg/2 mL (per mL): $960.00
500 mg/10 mL (per mL): $960.00
Disclaimer: A representative AWP (Average Wholesale Price) price or price range is provided as reference price only. A range is provided when more than one manufacturer's AWP price is available and uses the low and high price reported by the manufacturers to determine the range. The pricing data should be used for benchmarking purposes only, and as such should not be used alone to set or adjudicate any prices for reimbursement or purchasing functions or considered to be an exact price for a single product and/or manufacturer. Medi-Span expressly disclaims all warranties of any kind or nature, whether express or implied, and assumes no liability with respect to accuracy of price or price range data published in its solutions. In no event shall Medi-Span be liable for special, indirect, incidental, or consequential damages arising from use of price or price range data. Pricing data is updated monthly.
IV: Must be diluted prior to administration. Administer by IV infusion over 35 to 60 minutes through a 0.2-micron inline filter; infusion must be completed within 4 hours of preparation. Flush IV access line with NS prior to and after infusion; may consider application of a topical anesthetic cream to the infusion site prior to administration. Do not mix with other medications or infuse other medications concomitantly via the same IV access line. If a dose is missed, administer as soon as possible after the scheduled time.
IV: Administer by IV infusion over 35 to 60 minutes through a 0.2-micron inline filter; complete infusion within 4 hours of dilution. Consider slowing the infusion or interrupting therapy for hypersensitivity reactions. Flush IV access line with NS prior to and after infusion. Do not mix with other medications or infuse other medications concomitantly via the same IV access line.
Store at 2°C to 8°C (36°F to 46°F). Do not freeze. Protect from light and store in the original carton until ready for use. The diluted solution may be stored at 2°C to 8°C (36°F to 46°F) for up to 24 hours; do not freeze. Discard unused portion.
Treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping (FDA approved in male pediatric patients [age not specified] and adults)
Note: Indication approved based on a surrogate endpoint of an increase in dystrophin in skeletal muscle observed in some patients; a clinical benefit has not been established. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.
None known.
There are no known significant interactions.
Animal reproduction studies have not been conducted. Eteplirsen has not been studied in patients assigned female at birth.
Infusion site during infusion
Binds to exon 51 of dystrophin pre-messenger RNA (mRNA), resulting in exclusion of this exon during mRNA processing. Exon skipping allows for production of an internally truncated dystrophin protein.
Distribution: Vdss: 600 mL/kg
Protein binding: 6% to 17%
Half-life elimination: 3 to 4 hours
Time to peak: 1.1 to 1.2 hours
Excretion: Renal
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