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Eteplirsen(Exondys 51)

Acetylcysteine Injection, FLUIMUCIL, Acetylcysteine InjectionAcetylcysteine Injection, FLUIMUCIL, Acetylcysteine InjectionAcetylcysteine Injection, FLUIMUCIL, Acetylcysteine Injection

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Reminder: The outer packaging is for reference only, please purchase and use under the guidance of a pharmacist. For read by medical and pharmaceutical professionals only.

Eteplirsen is an antisense oligonucleotide designed to treat Duchenne muscular dystrophy (DMD), a severe genetic disorder caused by mutations in the dystrophin gene. By skipping exon 51 during mRNA processing, Eteplirsen enables the production of a shortened but functional dystrophin protein, which is crucial for muscle integrity.

The drug is administered intravenously on a weekly basis, offering a disease-modifying approach for eligible patients. Although not a cure, Eteplirsen has been shown to increase dystrophin levels, potentially slowing disease progression. Long-term efficacy and safety are under continuous study, reinforcing its role in DMD management

Generic name

Eteplirsen(Exondys 51)

English name

Exondys 51

Alternative Names

Eteplirsen

Drug prices

Indications

EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene amenable to exon 51 skipping. Approved under accelerated approval based on increased dystrophin production in skeletal muscle. Continued approval is contingent on verification of clinical benefit in confirmatory trials.

Therapeutic Target

Molecular Target: Exon 51 of dystrophin pre-mRNA.Mechanism: Binds to exon 51, inducing exon skipping during mRNA processing to produce truncated but functional dystrophin protein.

Active Ingredients

Eteplirsen (phosphorodiamidate morpholino oligomer [PMO] antisense oligonucleotide).

Dosage form

SOLUTION

specifications

100 mg/2 mL (50 mg/mL): NDC 60923-363-02. 500 mg/10 mL (50 mg/mL): NDC 60923-284-10.

Description

1.Physical Properties: Clear, colorless solution; may exhibit slight opalescence or trace white-to-off-white amorphous particles.

2.Formulation: Isotonic phosphate-buffered saline (pH 7.5).

3.Composition: Contains 50 mg/mL eteplirsen, potassium chloride, sodium chloride, and phosphate buffers.

Dosage and Administration

1.Dose: 30 mg/kg once weekly via intravenous infusion.

2.Preparation:

Dilute in 0.9% Sodium Chloride Injection to total volume of 100–150 mL.

Use in-line 0.2-micron filter.

3.Infusion: Administer over 35–60 minutes.

4.Missed Dose: Administer as soon as possible; do not double dose.

5.Storage of Diluted Solution: Use immediately or store at 2°C–8°C for ≤24 hours.

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