Eteplirsen
What is Eteplirsen?[edit | edit source]
- Eteplirsen (EXONDYS 51) is an antisense oligonucleotide used for the treatment of Duchenne muscular dystrophy (DMD).
What are the uses of this medicine?[edit | edit source]
- This medicine is used for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.
- This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51.
How does this medicine work?[edit | edit source]
- Eteplirsen is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping.
- Exon skipping is intended to allow for production of an internally truncated dystrophin protein, which was evaluated in Study 2 and Study 3.
Who Should Not Use this medicine ?[edit | edit source]
- This medicine have no usage limitations.
What drug interactions can this medicine cause?[edit | edit source]
- No clinically important drug interactions have been observed with EXONDYS 51.
Is this medicine FDA approved?[edit | edit source]
- It was approved for use in the United States in 2016.
How should this medicine be used?[edit | edit source]
Recommended Dosage:
- The recommended dose of EXONDYS 51 is 30 milligrams per kilogram administered once weekly as a 35 to 60 minute intravenous infusion via an in-line 0.2 micron filter.
Administration
- Dilution required prior to administration.
- Application of a topical anesthetic cream to the infusion site prior to administration of EXONDYS 51 may be considered.
- EXONDYS 51 is administered via intravenous infusion. Flush the intravenous access line with 0.9% Sodium Chloride Injection, USP, prior to and after infusion.
- Infuse the diluted EXONDYS 51 solution over 35 to 60 minutes via an in-line 0.2 micron filter. Do not mix other medications with EXONDYS 51 or infuse other medications concomitantly via the same intravenous access line.
- If a hypersensitivity reaction occurs, consider slowing the infusion or interrupting the EXONDYS 51 therapy
- If a dose of EXONDYS 51 is missed, it may be administered as soon as possible after the scheduled time.
What are the dosage forms and brand names of this medicine?[edit | edit source]
This medicine is available in fallowing doasage form: As Injection:
- 100 mg/2 mL (50 mg/mL) in single-dose vial
- 500 mg/10 mL (50 mg/mL) in single-dose vial
This medicine is available in fallowing brand namesː
- EXONDYS 51
What side effects can this medication cause?[edit | edit source]
The most common side effects of this medicine include:
- Balance disorder
- Vomiting
- Contact dermatitis
What special precautions should I follow?[edit | edit source]
- Hypersensitivity reactions, including rash and urticaria, pyrexia, flushing, cough, dyspnea, bronchospasm, and hypotension, have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy.
What to do in case of emergency/overdose?[edit | edit source]
- There is no experience with overdose of EXONDYS 51.
Can this medicine be used in pregnancy?[edit | edit source]
- There are no human or animal data available to assess the use of EXONDYS 51 during pregnancy.
Can this medicine be used in children?[edit | edit source]
- EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping, including pediatric patients.
What are the active and inactive ingredients in this medicine?[edit | edit source]
Active ingredient:
- eteplirsen
Inactive ingredients:
- sodium chloride
- potassium chloride
- potassium phosphate, monobasic
- sodium phosphate, dibasic, anhydrous
- sodium hydroxide
- hydrochloric acid
- water
Who manufactures and distributes this medicine?[edit | edit source]
- Packager: Sarepta Therapeutics, Inc.
What should I know about storage and disposal of this medication?[edit | edit source]
- Store EXONDYS 51 at 2°C to 8°C (36°F to 46°F).
- Do not freeze.
- Protect from light and store EXONDYS 51 in the original carton until ready for use.
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