Drug Insights

Is Golodirsen approved by the FDA?

26 June 2024
3 min read

Yes, golodirsen, marketed under the brand name Vyondys 53, is FDA approved. The U.S. Food and Drug Administration (FDA) approved golodirsen on December 12, 2019, for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.

What is Golodirsen?

Golodirsen is an antisense oligonucleotide used to treat Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness. It is specifically designed for patients who have a mutation in the DMD gene that is amenable to exon 53 skipping, which allows for the production of a functional dystrophin protein, albeit shorter than normal.

Dosage and Administration

Golodirsen is administered as an intravenous solution with a concentration of 50 mg/mL. The usual dose for both adults and children is 30 mg/kg, given once weekly through an intravenous infusion. The infusion process takes about 60 minutes to complete and must be performed by a healthcare provider.

Side Effects

Common Side Effects

  • Headache
  • Fever
  • Stomach pain
  • Falls
  • Cough
  • Runny or stuffy nose

Serious Side Effects

  • Pink, brown, or red urine
  • Foamy urine
  • Swelling in the face, hands, feet, or stomach

Signs of an allergic reaction, such as hives, itching, rash, blistering, peeling, fever, difficulty breathing, or swelling of the face, lips, tongue, or throat, require immediate medical attention.

Warnings and Precautions

  • Kidney Problems: Inform your doctor if you have a history of kidney issues. Golodirsen can affect kidney function, and regular monitoring through blood tests is necessary.
  • Pregnancy and Breastfeeding: The safety of golodirsen during pregnancy and breastfeeding has not been established. Patients should consult their doctor if they are pregnant or plan to become pregnant.

FDA Approval and Post-Approval Requirements

Golodirsen was approved under the FDA's "accelerated" approval pathway, which allows for earlier approval of drugs that treat serious conditions and fill an unmet medical need based on a surrogate endpoint. Continued approval for this indication is contingent upon verification of clinical benefit in confirmatory trials.

How is Golodirsen Given?

Golodirsen is given as a weekly infusion into a vein by a healthcare provider. It is crucial to follow all instructions and attend all scheduled appointments to ensure the medication's efficacy and safety.

Drug Interactions

Other medications, including prescription and over-the-counter medicines, vitamins, and herbal products, can interact with golodirsen. Patients should inform their doctor about all the medicines they are currently taking to avoid potential interactions.

Conclusion

Golodirsen (Vyondys 53) is an FDA-approved medication for the treatment of Duchenne muscular dystrophy in patients with a specific genetic mutation. Approved on December 12, 2019, it provides a targeted therapeutic option for managing this progressive and debilitating disease. As with any medication, it is essential to follow the prescribed dosage, be aware of potential side effects, and communicate with healthcare providers about all medications being taken.

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