drug · oligonucleotide
golodirsen
Marketedupdated 3mo agoby $SRPT Sarepta Therapeutics, Inc.🔗 ChEMBL
Approvedcommunity read0 votes
🐶 often sniffed alongside
top 1Oligonucleotide drug with a maximum clinical stage of Approval (across all indications), with an approval for duchenne muscular dystrophy and muscular dystrophy.
indications
investigational
Duchenne Muscular Dystrophy
targets
Dystrophin pre-mRNA
all catalysts(1)
Trials studying golodirsen
- phase4An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy
- —A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
- phase3An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
- phase2A 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.
Patent cliff
source: FDA Orange Bookfirst barrier falls
Dec 12, 2026
113 days
Earliest of all listed patents + FDA exclusivities. Generic / biosimilar entry typically only becomes possible AFTER both patent and exclusivity barriers expire.
patents (1)
- Jun 28, 2028formulationUS RE476911.9y
FDA exclusivities (1)
- Dec 12, 2026ODE-280Orphan Drug (7y)113d
News(5)
- Capricor CEO won’t rule out legal action against FDA after negative adcommAfter an FDA advisory committee voted 9-3 on Wednesday against recommending Capricor Therapeutics’ Duchenne muscular dystrophy cardiomyopathy drug for approval, stakeholders allege an “ulterior mot…biospace · 20d ago
- Capricor and Replimune prep for FDA adcomms, Q2 earnings roll on, Sarepta’s new leader, moreThe FDA published briefing documents ahead of advisory committee meetings for therapies from Capricor Therapeutics and Replimune indicate an uphill battle for the biotechs; GSK and AstraZeneca outl…biospace · 22d ago
- Sarepta names former AbbVie R&D exec Michael Severino as next CEOAfter leaving Tessera Therapeutics earlier this month, Michael Severino will take over Sarepta Therapeutics as the biotech awaits two key regulatory decisions in Duchenne muscular dystrophy.biospace · 24d ago
- Sarepta, in search of a turnaround, taps ex-AbbVie exec Severino as CEOMost recently the leader of buzzy startup Tessera Therapeutics, Severino will look to improve the fortunes of a company facing emerging competition and declining sales for its prized gene therapy E…biopharma_dive · 24d ago
- Dyne gets early 2027 decision date for Duchenne therapy, analysts expect ‘smooth’ reviewIf approved, Dyne Therapeutics’ zeleciment rostudirsen could “capture the majority” of the exon-51 Duchenne muscular dystrophy market, given its better efficacy and dosing profile versus Sarepta Th…biospace · 1mo ago
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