Eteplirsen

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 [ see Clinical Studies ( 14 ) ].

• 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. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51 [see Clinical Studies ( 14 )] .

• None.contraindicated

30 milligrams per kilogram of body weight once weekly ( 2.1 ) Administer as an intravenous infusion over 35 to 60 minutes via an in-line 0.2 micron filter ( 2.1 , 2.3 ) Dilution required prior to administration ( 2.2 ) 2.1 Dosing Information 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. If a dose of EXONDYS 51 is missed, it may be administered as soon as possible after the scheduled time. 2.2 Preparation Instructions EXONDYS 51 is supplied in single-dose vials as a preservative-free concentrated solution that requires dilution prior to administration. Parenteral drug products should be inspected visually for particulate matter and discoloration prior to administration, whenever solution and container permit. Use aseptic technique. Calculate the total dose of EXONDYS 51 to be administered based on the patient's weight and the recommended dose of 30 milligrams per kilogram. Determine the volume of EXONDYS 51 needed and the correct number of vials to supply the full calculated dose. Allow vials to warm to room temperature. Mix the contents of each vial by gently inverting 2 or 3 times. Do not shake. Visually inspect each vial of EXONDYS 51. EXONDYS 51 is a clear, colorless solution that may have some opalescence, and may contain white to off-white amorphous particles. Do not use if the solution in the vials is cloudy, discolored or contains extraneous particulate matter other than white to off-white amorphous particles.

Hypersensitivity Reactions: Hypersensitivity reactions, including bronchospasm, chest pain, cough, tachycardia, and urticaria, have occurred in patients treated with EXONDYS 51. If hypersensitivity reactions occur, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy. ( 2.3 , 5.1 ) 5.1 Hypersensitivity Reactions Hypersensitivity reactions, including bronchospasm, chest pain, cough, tachycardia, and urticaria, 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 [see Dosage and Administration ( 2.3 )] .

The most common adverse reactions (incidence ≥35% and higher than placebo) were balance disorder and vomiting ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc. at 1-888-SAREPTA (1-888-727-3782) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. EXONDYS 51 was studied in a double-blind, placebo-controlled study for 24 weeks (Study 1), followed by an open label extension (Study 2). In Study 1, 12 patients were randomized to receive weekly intravenous infusions of EXONDYS 51 (n=8) or placebo (n=4) for 24 weeks. All 12 patients continued in Study 2 and received open-label EXONDYS 51 weekly for up to 208 weeks. In Study 1, 4 patients received placebo, 4 patients received EXONDYS 51 30 mg/kg, and 4 patients received EXONDYS 51 50 mg/kg (1.7 times the recommended dosage). In Study 2, 6 patients received EXONDYS 51 30 mg/kg/week and 6 patients received EXONDYS 51 50 mg/kg/week [see Clinical Studies ( 14 )] . Adverse reactions that occurred in 2 or more patients who received EXONDYS 51 and were more frequent than in the placebo group in Study 1 are presented in Table 1 (the 30 and 50 mg/kg groups are pooled). Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice.

8.1 Pregnancy Risk Summary There are no human or animal data available to assess the use of EXONDYS 51 during pregnancy. In the U.S. general population, major birth defects occur in 2 to 4% and miscarriage occurs in 15 to 20% of clinically recognized pregnancies. 8.2 Lactation Risk Summary There are no human or animal data to assess the effect of EXONDYS 51 on milk production, the presence of eteplirsen in milk, or the effects of EXONDYS 51 on the breastfed infant. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for EXONDYS 51 and any potential adverse effects on the breastfed infant from EXONDYS 51 or from the underlying maternal condition. 8.4 Pediatric Use 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 [see Clinical Studies ( 14 )] . Intravenous administration of eteplirsen (0, 100, 300, or 900 mg/kg) to juvenile male rats once weekly for 10 weeks beginning on postnatal day 14 resulted in renal tubular necrosis at the highest dose tested and decreased bone densitometry parameters (mineral density, mineral content, area) at all doses.

Metabolism

Following single or multiple intravenous infusions of EXONDYS 51 in male pediatric DMD patients, plasma concentration-time profiles of eteplirsen were generally similar and showed multi-phasic decline. The majority of drug elimination occurred within 24 hours.

• Sep 19, 2016NDANDA206488Sarepta Theraps Inc

1. 1Product Dose Omission Issue65230%
2. 2No Adverse Event50323%
3. 3Intentional Dose Omission26012%
4. 4Poor Venous Access1627.4%
5. 5Device Issue1567.1%
6. 6Pyrexia904.1%
7. 7Covid-19853.9%
8. 8Fall592.7%
9. 9Malaise582.7%
10. 10Pneumonia582.7%
11. 11Cough542.5%
12. 12Influenza482.2%
13. 13Product Dose Omission411.9%
14. 14Weight Increased371.7%
15. 15Exposure To Sars-cov-2361.6%

The 10 most recently updated of 12 ClinicalTrials.gov registrations naming Eteplirsen as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
  Active not recruiting · Phase 3 · Interventional · 160 enrolled · Sarepta Therapeutics, Inc.
  NCT03992430updated 2026-02-27
• A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
  Enrolling by invitation · Observational · 300 enrolled · Sarepta Therapeutics, Inc.
  NCT06606340updated 2025-09-10
• A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.
  Completed · Phase 2 · Interventional · 3 enrolled · Kevin Flanigan
  NCT04179409updated 2023-10-26
• A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)
  Terminated · Phase 2 · Interventional · 15 enrolled · Sarepta Therapeutics, Inc.
  NCT03985878updated 2023-08-18
• Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping
  Completed · Phase 2 · Interventional · 15 enrolled · Sarepta Therapeutics, Inc.
  NCT03218995updated 2021-12-09
• Study of Eteplirsen in DMD Patients
  Completed · Phase 3 · Interventional · 109 enrolled · Sarepta Therapeutics, Inc.
  NCT02255552updated 2021-01-25
• Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
  Completed · Phase 2 · Interventional · 33 enrolled · Sarepta Therapeutics, Inc.
  NCT02420379updated 2021-01-25
• Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
  Completed · Phase 2 · Interventional · 24 enrolled · Sarepta Therapeutics, Inc.
  NCT02286947updated 2020-03-30
• Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients
  Completed · Phase 2 · Interventional · 12 enrolled · Sarepta Therapeutics, Inc.
  NCT01396239updated 2020-03-30
• Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy
  Completed · Phase 2 · Interventional · 12 enrolled · Sarepta Therapeutics, Inc.
  NCT01540409updated 2020-03-30

Frequently asked questions

How does Eteplirsen work?

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 [ see Clinical Studies ( 14 ) ].

What is Eteplirsen used for?

According to FDA labeling, Eteplirsen carries indications including: 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. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51 [see Clinical Studies ( 14 )] .. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Eteplirsen?

Eteplirsen is classified as Other drugs for disorders of the musculo-skeletal system, Antisense Oligonucleotide, Nucleic Acid Synthesis Inhibitors, Protein Synthesis Inhibitors, Decreased RNA Integrity.

What are the brand names for Eteplirsen?

Eteplirsen is marketed under brand names including Exondys.

What are the contraindications for Eteplirsen?

Eteplirsen labeling lists contraindications including: None.. Always consult the full prescribing information and a clinician.