• Brand Name: Exondys 51  
  • Active Ingredient: Eteplirsen
  • Strength: 100 mg/2 mL (50 mg/mL) & 500 mg/10 mL (50 mg/mL) injection
  • Originator Name/ Marketing-authorisation Holder: Sarepta Therapeutics

What is Exondys 51, and what it is used for:?


Exondys 51 is an FDA-approved treatment for Duchenne muscular dystrophy (DMD). It uses a technology named exon skipping to help the body make a shorter form of the dystrophin protein. This medicinal product is given in a once-weekly infusion.</p?

Dosage and Administration


The recommended dose is 30 mg/kg administered once weekly as a 35-60 minute intravenous infusion via an in-line 0.2 micron filter. If any dose of Exondys 51 is missed, it may be administered as quickly as possible after the scheduled time.

Use of a topical anesthetic cream to the infusion site prior to administration of injection may be considered.



Exondys 51 injection

is administered via intravenous infusion. Flush the intravenous access line with 0.9 percent Sodium Chloride Injection, before and after the infusion.


Infuse the diluted solution of Exondys 51 over 35 to 60 minutes through an in-line 0.2 micron filter.

Do not mix other medicines with Eteplirsen or infuse other medicines concomitantly via the same intravenous access line.


In case a hypersensitivity reaction occurs, slow the infusion or interrupt the therapy.


Side effects


As with any medicine, it’s necessary to talk to your health specialist about the probability of side effects with Exondys 51. Here’s what you should know about side effects experienced by those receiving this medicine in clinical studies.


Allergic reactions:

Allergic reactions have been noted in those treated with Exondys 51, including:


Chest pain



Rapid heart rate

Common side effects:

The commonly occurred side effects with Exondys 51 by intravenous infusion are:


  • Problems with balance
  • Vomiting
  • Skin irritation


Additional side effects:

The following side effects were seen in greater than 10 percent of patients who received Exondys 51:


  • Cough
  • Rash
  • Vomiting
  • Headache


Seek immediate medical help in case signs/symptoms of allergic reactions occur.


Precautions and Warnings


Hypersensitivity reactions, including rash and urticaria, flushing, pyrexia, cough, bronchospasm, dyspnea, and hypotension, may occur in those who received Exondys 51. In case a hypersensitivity reaction occurs, consider apt medical treatment and slow the infusion or interrupt the therapy.



Prior to receiving Exondys 51 injection, tell your health specialist If you are allergic to it; any part of this medication; or any other medicines, foods, or substances. Tell your health specialist about the allergy and what signs/symptoms you had.



Allergic reactions, including chest pain, cough, wheezing, rapid heart rate, and hives may occur in those who were received injection Exondys 51. Get immediate medical assistance in case signs/symptoms of allergic reactions occur.



Let your health specialist know in case you are pregnant or want/plan to become pregnant prior to using Exondys 51 treatment; it is not known if it would affect a fetus. It is not specified if this DMD medicine passes into human breast milk. Consult your health specialist prior to breastfeeding.


Handling and storage


The drug Exondys 51 is supplied in single-dose vials containing 100 mg/2 mL (50 mg/mL) eteplirsen, and 500 mg/10 mL (50 mg/mL) eteplirsen. The solution is clear and colorless, and may have some opalescence, and may contain trace amounts of tiny, white to off-white amorphous particles.



Store vials at 2°C to 8°C (36°F to 46°F). Freezing is not recommended. Protect from the light and store in the original carton.


Frequently asked questions (FAQs)

Exondys 51 injection is used for Duchenne muscular dystrophy (DMD) in those who have a confirmed mutation in the dystrophin gene that can be treated by skipping exon 51.

A genetic test is the only way for your child’s health specialist to identify your child’s mutation and determine if the mutation is amenable to treatment with Exondys 51. 

Weekly infusions of this medicinal product helped the body make a shorter form of the dystrophin protein in some recipients. 

Duchenne patients who receive this medicine must have a genetic test that demonstrates a mutation in the gene (dystrophin) that can be treated by skipping exon 51. Your child’s health specialist is best equipped to specify if your child’s mutation is amenable to treatment with this medicinal product.

Exondys 51 therapy is a weekly intravenous (IV) infusion that is always given by a health specialist. The medication is usually infused over 35 to 60 minutes. 

Infusions of Exondys 51 can be given at an infusion center, health specialist’s office, or your home. Discuss your options with your health specialist; there are certain factors to consider when determining what’s best for your family.

Talk to your health specialist. In case you miss a dose of Exondys 51, it may be administered as quickly as possible after the scheduled time.

You should discuss with your health specialist about all the medicine you are taking. Your health specialist is the best person to instruct you about your medications.

The amount of medication is based upon how much you weigh. The proposed dosage is 30 mg per kg of body weight, once weekly.

For medicine, Exondys 51 Sarepta Therapeutics is the innovator, and Ikris Pharma Network (IPN) is an authorized partner who can help access this medicine in India for the patient through legal procedures. To know more, contact IKRIS at TOLL-FREE: 18008891064 or Write at [email protected].

The Exondys 51 cost is reasonable. To authenticate this pharmaceutical medicinal product, you can Call/WhatsApp: +91 8130290915, Or dial TOLL-FREE: 18008891064, Or Email at: [email protected].

In India, you can buy Exondys 51 with the help of Ikris Pharma Network, an authorized partner of “Sarepta Therapeutics” for this pharmaceutical medicinal product.