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Vyondys 53 (GOLODIRSEN)

Sarepta · FDA-approved approved Oligonucleotide Quality 66/100

Vyondys 53 works by binding to specific RNA sequences to prevent the production of a faulty dystrophin protein.

Vyondys 53 (golodirsen) is an antisense oligonucleotide developed by Sarepta Therapeutics, targeting exon 53 of dystrophin pre-mRNA to treat Duchenne muscular dystrophy. It was FDA-approved in 2019 and remains a patented product with no generic manufacturers. Vyondys 53 works by binding to specific RNA sequences, preventing the production of a faulty dystrophin protein. Key safety considerations include its short half-life of 3.4 hours, and the potential for liver enzyme elevations. As a patented product, its commercial status is secure.

At a glance

Generic nameGOLODIRSEN
SponsorSarepta
Drug classAntisense Oligonucleotide [EPC]
Targetexon 53 of dystrophin pre-mRNA
ModalityOligonucleotide
Therapeutic areaRare Disease
PhaseFDA-approved
First approval2019
Annual revenue400

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)].

Approved indications

Common side effects

Key clinical trials

Patents

PatentExpiryType

Primary sources

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SourceUsed for
FDA labelMechanism, indications, dosing, boxed warnings, drug interactions
ClinicalTrials.govTrial enrolment, design, endpoints, results
FDA Orange BookPatents + exclusivity
SEC EDGARRevenue + earnings