VYONDYS 53

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.