• Brand Name: Vyondys 53
  • Active Ingredient: Golodirsen
  • Strength: 100 mg/2 mL (50 mg/mL) 
  • Originator Name/ Marketing-authorisation Holder: Sarepta Therapeutics



What is Vyondys 53, and what it is used for: ?

Vyondys 53 is an antisense oligonucleotide used for the treatment of patients with Duchenne muscular dystrophy who have a established mutation of the gene named DMD that is amenable to exon-53 skipping. Vyondys 53 (Golodirsen) treatment uses Sarepta’s proprietary PMO (Phosphorodiamidate Morpholino Oligomer) chemistry and an exon-skipping technology to bind to exon-53 of dystrophin pre-mRNA, resulting in exclusion, or skipping, of this exon during processing of mRNA in those with genetic mutations that are amenable to exon 53 skipping. Exon skipping is intended to allow for creation of an internally truncated dystrophin protein.

Dosage and Administration

Vyondys 53 is available/supplied in single-dose vials as a preservative-free concentrated solution that requires dilution before administration. Parenteral drug products needs to be inspected visually for particulate matter and discoloration before administration, whenever solution and container permit. Calculate the total dose to be administered on behalf of the patient’s weight and the recommended dose of 30 mg per kg. Determine the volume of medication needed and the appropriate number of vials to supply the full calculated dose. The recommended dosage of Vyondys 53 is 30 mg/kg administered once in week as a 35-60 minute intravenous infusion through an in-line 0.2 micron filter. If any dose is skipped/missed, it may be administered as quickly as possible after the scheduled dose. Use of a topical anesthetic cream to the infusion site prior to administration of injection Golodirsen may be considered.</p?

Side effects


Vyondys 53 can cause both mild or serious side effects. The following lists contain a few key side effects that may occur with Vyondys 53 therapy. These lists do not include all probable side effects.


Mild side effects:

Mild side effects with Vyondys 53 may include:


  • cough
  • falls
  • fever
  • headache
  • common cold
  • abdominal (belly) pain
  • nausea and vomiting


Often, these side effects may fade away usually within a few days or some weeks. But in case they become more severe or persist, talk with your health specialist.


Serious side effects:

Serious side effects with Vyondys 53 aren’t common, but can occur. Serious side effects may include:


  • allergic reaction
  • kidney damage
Precautions and Warnings


Hypersensitivity reactions, including pyrexia, rash, pruritus, dermatitis, urticaria, and skin exfoliation have occurred in those treated with Vyondys 53. In case a hypersensitivity reaction occurs, start appropriate medical treatment and slow the infusion or interrupt the therapy.



Urine dipstick, serum cystatin C, and urine protein-to-creatinine ratio need to be measured prior to starting treatment with Golodirsen injection. Consider measurement of glomerular filtration before initiating this treatment.



Monitoring for kidney toxicity throughout the treatment is required. Obtain the urine samples before the infusion of Vyondys 53 treatment or at least 48 hours following the most recent infusion.



Prior to taking Vyondys 53, talk with your health specialist about your health history. This medicine may not be appropriate for you in case you have certain medical conditions (such as kidney problems, allergic reaction), or other factors affecting your health.



It is unknown if it’s safe to use Vyondys 53 while pregnant. This is because golodirsen, the active ingredient in Vyondys 53, hasn’t been assessed during pregnancy. If you’re pregnant or planning a pregnancy, discuss with your health specialist about the risks/benefits of using this medicine.



It’s not known whether it’s safe to take Vyondys 53 injection while breastfeeding. This medication hasn’t been evaluated in connection with breastfeeding. Therefore it may cause side effects in a breastfed child. If you’re breastfeeding or want to breastfeed, discuss with your health specialist about the risks/benefits of using this DMD medicine.


Handling and storage


Vyondys 53 comes as a clear to slightly opalescent, colorless liquid. It may be a composition of trace amounts of small, white to off-white amorphous particles. It is available as Injection 100 mg/2 mL (50 mg/mL) solution in a single-dose vial.


Store at temperature 2°C to 8°C (36°F to 46°F). It is not advisable to freeze. Store in the original carton until the time of use in order to protect from the light.


Frequently asked questions (FAQs)

Vyondys 53 is a brand-name pharmaceutical prescription medicine. It’s approved by FDA to treat Duchenne muscular dystrophy (DMD) in those (adults and children) with a certain gene mutation.

Each mL of Vyondys 53 contains: 50 mg golodirsen; 0.2 mg potassium phosphate monobasic; 0.2 mg potassium chloride; 8 mg sodium chloride; and 1.14 mg sodium phosphate dibasic, anhydrous, in water for injection.

Vyondys 53 is supplied in 2 mL single-dose vials that contains 100 mg golodirsen (50 mg/mL). The solution is a clear-slightly opalescent, colorless liquid and may contain trace amounts of tiny, white to off-white amorphous particles. 

The amount of medicine you will be given depending bon your weight. The recommended dosage is 30 mg per kg of body weight, intravenously (IV) infused, once weekly over 35 to 60 minutes via in-line 0.2 micron filter. 

Vyondys 53 will be intravenously infused over 35 to 60 minutes with the help of an in-line 0.2 micron filter. 

If a dose is missed, it may be administered as quickly as possible after the scheduled dose. Discuss with your health specialist if you miss any dose of Vyondys 53.

For medication, Vyondys 53 Sarepta Therapeutics is the innovator, and Ikris Pharma Network is an authorized partner who can help access this medicine in India for the patient through legal procedures. To know more, contact IKRIS Pharma either via TOLL-FREE: 18008891064, Or E-mail: [email protected], Or Call/WhatsApp: +91 8130290915.

The Vyondys 53 cost in India is reasonable. In order to authenticate this pharmaceutical medicinal product, you can dial our TOLL-FREE: 18008891064, Or Call/WhatsApp: +91 8130290915, Or write about your requirement at: [email protected].

In India, you can buy Vyondys 53 with the help of Ikris Pharma Network (IPN), an authorized partner of “Sarepta Therapeutics” for this medicinal product.