drugset
Drugs / Exondys 51
last change Sep 2023 re-read 3 minutes ago

Exondys 51

targets DMD via restoration

Developed for
Duchenne muscular dystrophy
Investigated by
Kevin Flanigan · Sarepta Therapeutics, Inc.

Regulatory milestones approvals, filings & regulatory actions · 1 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Approved US (FDA) EXONDYS 51 — 2016-09-19 fda.gov ↗

Trials 1

today
US approved 2016
NCT04179409 · Ph 2
20172018201920202021202220232024202520262027
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 2 NCT04179409 Feb 2020 → Sep 2021 Duchenne muscular dystrophy Kevin Flanigan Completed No outcome recorded

Press releases naming this drug 2 releases

DateIssuerRelease
2018-01-08 Sarepta Therapeutics, Inc. Sarepta Therapeutics Pre-Announces Fourth Quarter 2017 Revenue and Provides Full-Year 2018 Revenue Guidance for EXONDYS 51® (eteplirsen), Representing Approximately 100 Percent Year-over-Year Growth sarepta.com ↗
2016-09-19 Sarepta Therapeutics, Inc. Sarepta Therapeutics Announces FDA Accelerated Approval of EXONDYS 51™ (eteplirsen) injection, an Exon Skipping Therapy to Treat Duchenne Muscular Dystrophy (DMD) Patients Amenable to Skipping Exon 51 sarepta.com ↗

Evidence & citations 3 cited values

Every value below carries the sentence it was read from. 1 source stands behind the page.

FieldValueCited text
Known as Exondys 51 ClinicalTrials.gov intervention name — accepted as the source's own label NCT04179409 ↗
Action Restore “Skipping of a single copy of the duplicated exon is expected to result in a wild-type (WT) DMD transcript allowing the expression of a WT, full length dystrophin protein.” NCT04179409 ↗
Target DMD “This drug is used to target skipping of exon 51 of the dystrophin gene.” NCT04179409 ↗