GOLODIRSEN

Other

Brand names: Vyondys 53

Intravenous

Mechanism of Action

12.1 Mechanism of Action Golodirsen is designed to bind to exon 53 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon 53 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping [see Clinical Studies ( 14 )].

Indications

See full prescribing information

Contraindications

• 5.1 )

FDA Warnings

5 WARNINGS AND PRECAUTIONS Hypersensitivity Reactions: Hypersensitivity reactions, including anaphylaxis, rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation have occurred in patients who were treated with VYONDYS 53
VYONDYS 53 is contraindicated in patients with a history of a serious hypersensitivity reaction to golodirsen or to any of the inactive ingredients in VYONDYS 53 [see Contraindications ( 4 )]
Although kidney toxicity was not observed in the clinical studies with VYONDYS 53, the clinical experience with VYONDYS 53 is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides

Known Side Effects

5 documented side effects by frequency

Fever

0.1-1%

General Unwellness

0.1-1%

Vomiting

0.1-1%

Nausea

0.1-1%

Rash

0.1-1%

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Formulations

Dosage Forms

Tablet

Route

Intravenous