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vyondys 53

Generic: GOLODIRSEN

vyondys 53: developed by 1 company · 1 clinical trial tracked · 1 FDA decision.

vyondys 53 evidence translation

What we can confirm

1 FDA record · 1 tracked trial

What it means

Source-backed records connect vyondys 53 to SRPT; that link alone does not prove the drug works.

What to watch

Watch active trials and any change in FDA or trial status.
BioSniper AIPage evidence connected

Prepared research question

How could vyondys 53 → SRPT affect its developer, and what evidence supports the link?

Run cited research

Traceable citations · Unknowns marked

See linked entities and source evidence1 indications · 0 study sponsors

Related indications

Study sponsors

No additional listed-company sponsor is linked.

Development Pipeline (1)
CompanyPhase
Sarepta Therapeutics, Inc. (SRPT)Approved
Clinical Trials (1)

A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.

NCT04179409·Phase 2
Completed

Primary endpoint: Change in Dystrophin Expression From Baseline Following Treatment With Either AMONDYS 45 (Previously Casimersen), EXONDYS 51 (Previously Eteplirsen ), or VYONDYS 53 (Previously Golodirsen)

Interventionalopen3 enrolledStarted 2020-02Completion 2023-091 site · United StatesKevin Flanigan
FDA Decisions1 records
Approval1 INDICATIONS AND USAGE VYONDYS 53 is indicated 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. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53 [see Clinical Studies ( 14 )] . Continued approval for this indication may be contingent upon verification of a clinical benefit in conf(SRPT)FDA ↗
NDA211970Type 1 - New Molecular EntityOrphan DrugPriorityIntravenousSolution

Frequently asked questions

Who is developing vyondys 53?

vyondys 53 is being developed by Sarepta Therapeutics, Inc..

How many clinical trials involve vyondys 53?

BioSniper tracks 1 clinical trial involving vyondys 53.

What is vyondys 53's latest FDA decision?

The most recent tracked FDA decision for vyondys 53 is Approval for 1 INDICATIONS AND USAGE VYONDYS 53 is indicated 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. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53 [see Clinical Studies ( 14 )] . Continued approval for this indication may be contingent upon verification of a clinical benefit in conf, dated December 12, 2019.