Golodirsen

Golodirsen is a prescription medication used to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping.

• Golodirsen is available under the following different brand names: Vyondys 53

Common side effects of Golodirsen include:

• headache
• fever
• falls
• abdominal pain
• runny or stuffy nose
• cough
• vomiting
• nausea
• administration site pain
• back pain
• diarrhea
• dizziness
• ligament sprain
• contusion
• influenza
• mouth and throat pain
• skin abrasion
• ear infection
• seasonal allergy
• fast heart rate
• catheter site-related reaction
• constipation
• fracture

Serious side effects of Golodirsen include:

• hives
• itching
• rash
• blistering or peeling
• fever
• difficult breathing
• swelling of the face, lips, tongue, or throat
• pink, brown, or red urine
• foamy urine
• swelling in the face, hands, feet, or stomach

Rare side effects of Golodirsen include:

• none

Seek medical care or call 911 at once if you have the following serious side effects:

• Severe headache, confusion, slurred speech, arm or leg weakness, trouble walking, coordination loss, unsteady, very stiff muscles, high fever, profuse sweating, or tremors;
• Serious eye symptoms such as sudden vision loss, blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights;
• Serious heart symptoms include fast, irregular, or pounding heartbeats; fluttering in the chest; shortness of breath; sudden dizziness, lightheadedness, or passing out.

This is not a complete list of side effects and other serious side effects or health problems that may occur because of the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may report side effects or health problems to FDA at 1-800-FDA-1088.

Adult and pediatric dosage

Injection, concentrated solution

• 100 mg/2 mL (50 mg/mL) single-dose vial

Duchenne muscular dystrophy

Adult and pediatric dosage

• 30 mg/kg IV every week

Dosage Considerations – Should be Given as Follows:

• See “Dosages”

If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, health care provider, or pharmacist first.

• Golodirsen has no noted severe interactions with any other drugs
• Golodirsen has no noted serious interactions with any other drugs
• Golodirsen has no noted moderate interactions with any other drugs
• Golodirsen has no noted minor interactions with any other drugs

This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all the products you use. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your healthcare professional or doctor for additional medical advice, health questions, or concerns.

Contraindications

• None

Effects of drug abuse

• None

Short-Term Effects

• See “What Are Side Effects Associated with Using Golodirsen?”

Long-Term Effects

• See “What Are Side Effects Associated with Using Golodirsen?”

Cautions

• Hypersensitivity
  • Hypersensitivity reactions, including rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation, were reported, some requiring treatment
  • If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting therapy
• Renal toxicity
  • Renal toxicity was observed in animals
  • Although renal toxicity was not observed in the clinical studies, renal toxicity, including potentially fatal glomerulonephritis, has been observed after the administration of some antisense oligonucleotides
• Monitor renal function during therapy
  • Because of the effect of reduced skeletal muscle mass on creatinine measurements, serum creatinine may not be a reliable measure of renal function in patients with DMD
  • Measure glomerular filtration rate (GFR) by 24-hour urine collection before initiating therapy
  • Monthly monitoring for proteinuria by dipstick urinalysis and monitoring of serum cystatin C and urine protein-to-creatinine ratio every 3 months is recommended
  • If confirmed dipstick proteinuria of 2+ or greater or elevated serum cystatin C, a 24-hour urine collection to quantify proteinuria and assess GFR should be performed
  • Only urine expected to be free of the excreted drug should be used for monitoring of urine protein; urine obtained on the day of infusion before infusion, or urine obtained at least 48 hours after the most recent infusion, may be used; alternatively, use a laboratory test that does not use the reagent pyrogallol red, as this reagent has the potential to cross-react with drug excreted in the urine and thus lead to a false-positive result for urine protein
  • If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation

Pregnancy and Lactation

• No human or animal data are available to assess use during pregnancy
• Lactation
  • No human or animal data are available to assess the drug’s effects on milk production, presence in milk, or effects on the breastfed infant