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.
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.
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.
Common Side Effects
Serious Side Effects
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.
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.
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.
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.
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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