WHAT IS VYONDYS 53 (golodirsen)?
VYONDYS 53 is used to treat patients with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the dystrophin gene that can be treated by skipping exon 53. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.
Who can take VYONDYS 53?
Patients who receive VYONDYS 53 must have a genetic test that shows a mutation in the dystrophin gene that can be treated by skipping exon 53. A healthcare provider is needed to interpret your genetic test to determine whether you can take VYONDYS 53.
What are the most common side effects reported with VYONDYS 53?
Adverse reactions that have occurred in at least 20% of patients treated with VYONDYS 53 and more often than in patients who received an inactive intravenous (IV) infusion were headache (41%, 10%), fever (41%, 14%), fall (29%, 19%), pain in the abdomen (27%, 10%), infection of the nose and throat (27%, 14%), cough (27%, 19%), vomiting (27%, 19%), and nausea (20%, 10%).
Should I continue taking my other medications while on VYONDYS 53?
You should talk with your doctor about all the medications you are taking. Your doctor is the best person to advise you about your medicines.
How is VYONDYS 53 supplied?
VYONDYS 53 is supplied in 2 mL single-dose vials containing 100 mg golodirsen (50 mg/mL).
How much VYONDYS 53 will I receive?
The amount of VYONDYS 53 you will be given is based upon how much you weigh. The recommended dosage of VYONDYS 53 is 30 milligrams per kilogram of body weight, once weekly over 35-60 minutes.
How long will my infusion last?
VYONDYS 53 will be intravenously infused over 35–60 minutes.
What happens if I miss an infusion?
If a dose of VYONDYS 53 is missed, it may be administered as soon as possible after the scheduled dose. Talk to your doctor if you miss a dose.
Where will I get my infusion?
You may receive your infusions at an infusion center or your home. You and your doctor may need to discuss these options, including whether home therapy is an option for you.
How is VYONDYS 53 administered?
VYONDYS 53 is given by intravenous (IV) infusion once a week. An IV infusion is a way of delivering medicine directly into your bloodstream through a vein. Your doctor may discuss the use of a port, which is a device installed under the skin for repeat use in delivering IV medications. VYONDYS 53 infusion is always given and monitored by a healthcare provider.
Are there any special considerations when using a port?*
Ask your doctor for any patient instructions provided by the maker of your port. Carefully follow these or other instructions provided by your doctor for care of your port site to reduce the risk of complications, including infections.
What kind of care is required when using a port?*
Your doctor or the maker of your port may recommend hygiene measures after placement of the port and after each use of the port. Carefully follow these and other instructions provided by your doctor.
When should I contact my doctor about my port?*
Follow instructions from the maker of your port and your doctor regarding when to contact your doctor. Always contact your doctor:
- If you notice any redness, tenderness, bruising, swelling, warmth or drainage at or near the injection site
- For a fever
- If there is swelling, tingling or pain at or near the port injection site or in the arm closest to the port.
*Always refer to the manufacturers instruction for use (IFU) guide for more information on safety and precautions and ask your health care provider to review the relevant instruction for use of your port with you.
POSSIBLE SIDE EFFECTS OF VYONDYS 53
Allergic reactions, including rash, fever, itching, hives, and inflammation and/or peeling of the skin have occurred in patients who were treated with VYONDYS 53. Seek immediate medical care if signs and symptoms of allergic reactions occur.
Damage to the kidneys was seen in animals who received golodirsen. Although damage to the kidneys was not seen in clinical studies with VYONDYS 53, potentially fatal kidney damage has occurred with other drugs that work in a similar way. Your doctor may recommend urine collection and blood testing before starting treatment followed by urine testing every month and a blood test every 3 months to monitor your kidneys.
Table 1: Adverse Reactions That Occurred in At Least 20% of VYONDYS 53-Treated Patients and at a Rate Greater than Placebo in Studies 1 and 2
| Adverse Reaction | VYONDYS 53 (N = 41) % |
Placebo (N = 21) % |
|---|---|---|
| Headache | 41 | 10 |
| Pyrexia | 41 | 14 |
| Fall | 29 | 19 |
| Abdominal pain | 27 | 10 |
| Nasopharyngitis | 27 | 14 |
| Cough | 27 | 19 |
| Vomiting | 27 | 19 |
| Nausea | 20 | 10 |
Other adverse reactions that occurred in greater than 5% of patients treated with VYONDYS 53 and more often than in patients who received an inactive IV infusion were pain at the IV site, back pain, pain, diarrhea, dizziness, stretch or tear in a ligament, bruising, flu, pain in the mouth and throat, stuffy or runny nose, scrapes or scratches of the skin, ear infection, seasonal allergy, fast heartbeat, reactions related to the IV catheter site, constipation, and broken bones. Talk to your doctor if you experience any side effects.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782)
Please see the full Prescribing Information for VYONDYS 53 (golodirsen).
A genetic test is required to confirm that a patient's mutation of the DMD gene is amenable to exon 53 skipping. For more information about genetic testing resources, visit Parent Project Muscular Dystrophy’s (PPMD) Decode Duchenne website.
