Exondys 51

Exondys 51 Uses, Dosage, Side Effects, Food Interaction and all others data.

Exondys 51 is a phosphoramidite morpholino sequence complementary to a portion of exon 51. It exerts it's mechanism of action by forcing the exclusion of exon 51 from the mature DMD mRNA.

The defining characteristic of DMD is lack of dystrophin, which is a protein that plays a vital role in maintaining muscle cell membrane integrity. (2) This is caused by a mutation in the DMD gene which leads to disruption of the translational reading frame, and ultimately in a non-functional protein. (2) Exondys 51, is a targeted oligonucleotide that causes exon skipping of exon 51 and restores the translational reading frame. (2) The expected result is production of an internally deleted but functional dystrophin protein. (2)Three clinical studies were done to evaluate eteplirsen. All patients in studies had a confirmed mutation of the DMD gene susceptible to exon 51 skipping.All patients treated with the drug produced messenger ribonucleic acid (mRNA) coding for an internally truncated dystrophin protein.In study 3, after 48 weeks of treatment with eteplirsen, the average dystrophin protein level was 0.44% of normal (what would be found in a healthy subject) compared to 0.16% of normal prior to treatment. There was a median increase of 0.1% in truncated dystrophin.

Trade Name Exondys 51
Availability Prescription only
Generic Eteplirsen
Eteplirsen Other Names Eteplirsen
Related Drugs deflazacort, Emflaza, Exondys 51, Amondys 45, Vyondys 53, golodirsen
Weight 50mg/ml,
Type Intravenous solution
Protein binding

Studies suggest that plasma protein binding of eteplirsen is 6-17% in humans.

Groups Approved, Investigational
Therapeutic Class
Available Country United States
Last Updated: September 19, 2023 at 7:00 am
Exondys 51
Exondys 51


Exondys 51 is an antisense oligonucleotide used to treat Duchenne muscular dystrophy (DMD) only in patients who are confirmed to have a specific type of mutation.

Exondys 51 is indicated for treatment of certain individuals with Duchenne muscular dystrophy (DMD). Its use is limited to those with a confirmed mutation of the DMD gene which would benefit from exon 51 skipping. Based on clinical studies showing increased dystrophin production in skeletal muscle in some patients given this drug, the above indication was approved under accelerated approval. Further confirmatory trials are required to demonstrate clinical benefit of eteplirsen.

Exondys 51 is also used to associated treatment for these conditions: Duchenne's Muscular Dystrophy (DMD)

How Exondys 51 works

Exondys 51, is a targeted oligonucleotide that causes exon skipping of exon 51 and restores the translational reading frame. (2) The expected result is production of an internally deleted but functional dystrophin protein. (2)


Potential adverse effects of eteplirsen were determined in 2 studies: 1. A 24 week double-blind, placebo-controlled study, 2. An open label extension following the first study. In study 1, the following adverse effects occurred more frequently in 2 or more patients receiving eteplirsen compared to placebo: Balance disorder (38%), vomiting (38%), contact dermatitis (25%). These percentages represent crude frequencies due to the small sample of patients studied; therefore, they may not be representative of the general population. All patients in study 1 continued to participate in study 2. The following adverse effects occurred at a rate ≥10% among the 88 patients receiving ≥30 mg/kg/week of eteplirsen for up to 208 weeks in study 2: vomiting, contusion, excoriation, arthralgia, rash, pain at catheter site, and upper respiratory tract infections. These adverse events also occurred more frequently in patients in study 2 compared to patients in study 1 on the same dose of eteplirsen. Facial flushing, transient erythema, and elevated temperature have been reported to occur on the days of eteplirsen infusion.

Food Interaction

No interactions found.

Volume of Distribution

Mean apparent volume of distribution = 600 mL/kg (value found following weekly intravenous infusion at a dose of 30mg/kg)

Half Life

Mean half-life (dose of 30 mg/kg) = 3.3 hours, and mean half-life (dose of 50 mg/kg) = 3.2 hours (3)


The kidneys are responsible for 65-70% of total eteplirsen clearance. (3) After 12 weeks of treatment at a dose of 30 mg/kg/week, total clearance was found to be 339 mL/hr/kg.

Elimination Route

Two-thirds of eteplirsen is renally eliminated within 24 hours of intravenous administration.

Innovators Monograph

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