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eteplirsen

Generic: ETEPLIRSEN·Brand: EXONDYS 51, Exondys

eteplirsen: developed by 1 company · 3 clinical trials tracked · 1 FDA decision.

eteplirsen evidence translation

What we can confirm

1 FDA record · 3 tracked trials

What it means

Source-backed records connect eteplirsen 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 eteplirsen → SRPT affect its developer, and what evidence supports the link?

Run cited research

Traceable citations · Unknowns marked

See linked entities and source evidence3 indications · 0 study sponsors

Related indications

Duchenne muscular dystrophy in patients with confirmed mutation of the dystrophin gene amenable to exon 51 skippingMuscular Dystrophy, Duchenne; Duchenne Muscular DystrophyDuchenne Muscular Dystrophy

Study sponsors

No additional listed-company sponsor is linked.

Development Pipeline (3)
CompanyPhase
Sarepta Therapeutics, Inc. (SRPT)
Duchenne muscular dystrophy in patients with confirmed mutat…
Approved
Sarepta Therapeutics, Inc. (SRPT)
Muscular Dystrophy, Duchenne; Duchenne Muscular Dystrophy
Phase 3
Sarepta Therapeutics, Inc. (SRPT)Approved
Clinical Trials (3)

A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of High Doses of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients With Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping

NCT03992430·Phase 3·SRPT
Active, not recruiting

Primary endpoint: Part 1: Incidence of Adverse Events (AEs)

InterventionalRandomizedquadruple160 enrolledStarted 2020-07Completion 2026-1050 sites · Colombia, Czechia, Denmark, France…Sarepta Therapeutics, Inc.

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

A Long-term Observational Study Evaluating Sarepta Therapeutics, Inc.'s Exon-Skipping Therapies in Patients With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice

NCT06606340·SRPT
Enrolling by invitation

Primary endpoint: Loss of Ambulation (LOA)

Observational300 targetStarted 2019-01Completion 2033-1220 sites · United StatesSarepta Therapeutics, Inc.
FDA Decisions1 records
Approval1 INDICATIONS AND USAGE EXONDYS 51 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 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51 [see Clinical Studies ( 14 )] . Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmato(SRPT)FDA ↗
NDA206488Type 1 - New Molecular EntityOrphan DrugPriorityIntravenousSolution

Frequently asked questions

Who is developing eteplirsen?

eteplirsen is being developed by Sarepta Therapeutics, Inc..

How many clinical trials involve eteplirsen?

BioSniper tracks 3 clinical trials involving eteplirsen.

What is eteplirsen's latest FDA decision?

The most recent tracked FDA decision for eteplirsen is Approval for 1 INDICATIONS AND USAGE EXONDYS 51 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 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51 [see Clinical Studies ( 14 )] . Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmato, dated September 19, 2016.