The indication for golodirsen is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with the drug. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.
- TRADE NAME: Vyondys 53 (Sarepta Therapeutics)
- INDICATIONS: Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.
- CLASS: Antisense oligomer
- HALF-LIFE: 3 hours
- FDA APPROVAL DATE: 12/12/2019
- CLINICALLY IMPORTANT, POTENTIALLY HAZARDOUS INTERACTIONS WITH:
None known - PREGNANCY: There are no human or animal data available to assess the use of golodirsen during pregnancy.
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Page last updated 07/31/2023
