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Synonyms: SRP-4053 | SRP4053 | VYONDYS 53®
golodirsen is an approved drug (FDA (2019))
Compound class:
Nucleic acid
Comment: Golodirsen (SRP-4053) is an antisense RNA oligonucleotide that uses the phosphorodiamidate morpholino oligomer (PMO) chemistry [2]. It was designed to induce exon 53 skipping in the dystrophin gene as a mechanism to increase expression of functional dystrophin protein in the muscle fibers of appropriately selected Duchenne muscular dystrophy patients [1].
Full sequence as provided in the agent's INN record is: all-P-ambo-[2′,3′-Azanediyl-P-(dimethylamino)-P,2′,3′-trideoxy-2′,3′-seco](2′-N→5′)(G-T-T-G-C-C-T-C-C-G-G-T-T-C-T-G-A-A-G-G-T-G-T-T-C) 5′-{P-[4-({2-[2-(2-hydroxyethoxy)ethoxy]ethoxy}carbonyl)piperazin-1-yl]-N,N-dimethylphosphonamidate}, but we have been unable to resolve this to a SMILES or HELM notation. |
| No information available. |
Summary of Clinical Use  |
| Golodirsen was approved by the FDA in December 2019, via their accelerated approval route, to treat Duchenne muscular dystrophy (DMD) patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. Such mutations are estimated to be present in ~8% of DMD patients [1]. |
| Clinical Trials | |||||
| Clinical Trial ID | Title | Type | Source | Comment | References |
| NCT02500381 | Study of SRP-4045 and SRP-4053 in DMD Patients | Phase 3 Interventional | Sarepta Therapeutics, Inc. | ||
| NCT04179409 | A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD Duplications | Phase 2 Interventional | Nationwide Children's Hospital | ||
| External links |
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For extended ADME data see the following: Drugs.com |
