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| Stem definition | Drug id | CAS RN |
|---|---|---|
| antisense oligonucleotides | 5357 | 1422959-91-8 |
| Molecule | Description |
|---|---|
|
Synonyms:
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Golodirsen is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) subclass. It is designed to bind to exon 53 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon 53 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping.
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| Date | Agency | Company | Orphan |
|---|---|---|---|
| Dec. 12, 2019 | FDA | SAREPTA THERAPS INC |
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| MedDRA adverse event term | Likelihood ratio | Likelihood ratio threshold | Patients taking drug having adverse event | Patients taking drug not having adverse event | Patients not taking drug having adverse event | Patients not taking drug not having adverse event |
|---|---|---|---|---|---|---|
| Product dose omission issue | 355.64 | 75.44 | 112 | 427 | 154253 | 42466543 |
| Poor venous access | 319.40 | 75.44 | 58 | 481 | 7194 | 42613602 |
| No adverse event | 271.87 | 75.44 | 68 | 471 | 38953 | 42581843 |
| Intentional dose omission | 149.57 | 75.44 | 32 | 507 | 8954 | 42611842 |
| Device issue | 105.45 | 75.44 | 26 | 513 | 13483 | 42607313 |
| MedDRA adverse event term | Likelihood ratio | Likelihood ratio threshold | Patients taking drug having adverse event | Patients taking drug not having adverse event | Patients not taking drug having adverse event | Patients not taking drug not having adverse event |
|---|---|---|---|---|---|---|
| Product dose omission issue | 396.47 | 95.52 | 110 | 328 | 321672 | 110267189 |
| No adverse event | 291.58 | 95.52 | 65 | 373 | 73774 | 110515087 |
| Poor venous access | 271.62 | 95.52 | 51 | 387 | 24471 | 110564390 |
| Intentional dose omission | 155.13 | 95.52 | 31 | 407 | 20049 | 110568812 |
| Device issue | 100.82 | 95.52 | 24 | 414 | 34511 | 110554350 |
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| Source | Code | Description |
|---|---|---|
| ATC | M09AX08 | MUSCULO-SKELETAL SYSTEM OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM Other drugs for disorders of the musculo-skeletal system |
| MeSH PA | D015335 | Molecular Probes |
| MeSH PA | D016376 | Oligonucleotides, Antisense |
| FDA CS | M0025055 | Oligonucleotides, Antisense |
| FDA PE | N0000009533 | Increased Protein Synthesis |
| FDA EPC | N0000191626 | Antisense Oligonucleotide |
| Disease | Relation | SNOMED_ID | DOID |
|---|---|---|---|
| Duchenne muscular dystrophy | indication | 76670001 | DOID:11723 |
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| Formulation strength | Trade name | Applicant | Application number | Approval date | Type | Dose form | Route | Exclusivity date | Description |
|---|---|---|---|---|---|---|---|---|---|
| 100MG/2ML (50MG/ML) | VYONDYS 53 | SAREPTA THERAPS INC | N211970 | Dec. 12, 2019 | RX | SOLUTION | INTRAVENOUS | Dec. 12, 2026 | 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 53 SKIPPING |
| Target | Class | Pharos | UniProt | Action | Type | Activity value (-log[M]) | Mechanism action | Bioact source | MoA source |
|---|---|---|---|---|---|---|---|---|---|
| exon 53 of dystrophin pre-mRNA | RNA | ANTISENSE INHIBITOR | UNKNOWN | DRUG LABEL |
| ID | Source |
|---|---|
| 4039025 | VANDF |
| CHEBI:757211 | CHEBI |
| CHEMBL4297762 | ChEMBL_ID |
| D11707 | KEGG_DRUG |
| C000710673 | MESH_SUPPLEMENTAL_RECORD_UI |
| 13605 | IUPHAR_LIGAND_ID |
| DB15593 | DRUGBANK_ID |
| 838470000 | SNOMEDCT_US |
| 838482009 | SNOMEDCT_US |
| C5222004 | UMLSCUI |
| 10355 | INN_ID |
| 328504 | MMSL |
| 37857 | MMSL |
| d09468 | MMSL |
| 018234 | NDDF |
| 033072U4MZ | UNII |
| 2267207 | RXNORM |
| Product | Category | Ingredients | NDC | Form | Quantity | Route | Marketing | Label |
|---|---|---|---|---|---|---|---|---|
| Vyondys 53 | HUMAN PRESCRIPTION DRUG LABEL | 1 | 60923-465 | INJECTION | 50 mg | INTRAVENOUS | NDA | 26 sections |