golodirsen
Trade name: Vyondys 53
Approved
Dec 12, 2019
Golodirsen, sold under the brand name Vyondys 53, is a medication used for the treatment of Duchenne muscular dystrophy. It is an antisense oligonucleotide medication of phosphorodiamidate morpholino oligomer (PMO) chemistry. — Wikipedia
Clinical trial activity
5 trials · 8 clinical orgs · 1 marketing orgs
Earliest trial started Dec 1, 2014 (NCT02310906)
Timeline
2010s
- Jan 1, 2014
Sarepta Therapeutics — Earliest Phase 1 Sponsor(trial)
- Jan 1, 2016
Sarepta Therapeutics — Earliest Phase 3 Sponsor(trial)
- May 22, 2018
Orphan Drug Designation
- Dec 12, 2019
Sarepta Therapeutics — Earliest FDA Approval
- Dec 12, 2019
Sarepta Therapeutics — Marketing Organization
- Dec 12, 2019
Sarepta Therapeutics — NDA Organization
2020s
- Jan 1, 2020
Earliest Phase 2 Sponsor(trial)
Indications
Studied for
Mechanism of action
- Dystrophin pre-mRNAACTIVATOR
Dystrophin pre-mRNA activator
- Dystrophin pre-mRNAPOSITIVE MODULATOR
Dystrophin pre-mRNA positive modulator
In Duchenne muscular dystrophy, deletion of exon 53 prevents synthesis of a functional dystrophin protein. Golodirsen is a 'molecular patch' that allows skipping of exon 53 and the assembly of remaining exons (54 onwards) to produce an internally truncated but functional dystrophin protein (that is missing exons 52/53).
Approval history
- approvedPriority reviewAcceleratedFast trackDec 12, 2019
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Delivery
- Parenteral
- Availability
- Prescription Only
Oral
No
Parenteral
Yes
Topical
No
Sources
- WikipediaGolodirsen ↗
- NCATS033072U4MZ ↗
- ChEMBLCHEMBL4297762 ↗
Also known as
- srp-4053
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT04708314 | Phase 4 | Oct 31, 2020 | Rare Disease Therapeutics, Sarepta Therapeutics |
| NCT04179409 | Phase 2 | Jan 1, 2020 | Ohio State University, Sarepta Therapeutics |
| NCT03532542 | Phase 3 | Aug 2, 2018 | Sarepta Therapeutics |
| NCT02500381 | Phase 3 | Aug 1, 2016 | Sarepta Therapeutics |
| NCT02310906 | Phase 1/Phase 2 (Phase 2) | Dec 1, 2014 | Catholic University of Sacred Heart, Rome, Institut de Myologie, France, Newcastle University, Royal Holloway University, Sarepta Therapeutics, University of London |
Organizations
Research & Development (8)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Sarepta Therapeutics | For profit | 5 | 3 | 3 | 2014 |
| Catholic University of Sacred Heart, Rome | Academic/Hospital | 1 | 0 | 1 | 2014 |
| Institut de Myologie, France | Academic/Hospital | 1 | 0 | 1 | 2014 |
| Newcastle University | Academic/Hospital | 1 | 0 | 1 | 2014 |
| Ohio State University | Academic/Hospital | 1 | 1 | 1 | 2020 |
| Rare Disease Therapeutics | For profit | 1 | 1 | 1 | 2020 |
| Royal Holloway University | Academic/Hospital | 1 | 0 | 1 | 2014 |
| University of London | Academic/Hospital | 1 | 0 | 1 | 2014 |
Marketing (2)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| Sarepta Therapeutics | For profit | MKTG | Dec 12, 2019 |
| Sarepta Therapeutics | For profit | NDA | Dec 12, 2019 |