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| Stem definition | Drug id | CAS RN |
|---|---|---|
| antisense oligonucleotides | 5182 | 1173755-55-9 |
| Molecule | Description |
|---|---|
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Synonyms:
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Eteplirsen is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein.
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| Date | Agency | Company | Orphan |
|---|---|---|---|
| Sept. 19, 2016 | FDA | SAREPTA THERAPEUTICS 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 |
|---|---|---|---|---|---|---|
| No adverse event | 1993.45 | 69.58 | 471 | 2529 | 38550 | 42579785 |
| Product dose omission issue | 1884.21 | 69.58 | 601 | 2399 | 153764 | 42464571 |
| Intentional dose omission | 1195.74 | 69.58 | 240 | 2760 | 8746 | 42609589 |
| Poor venous access | 644.12 | 69.58 | 139 | 2861 | 7113 | 42611222 |
| Device issue | 543.01 | 69.58 | 136 | 2864 | 13373 | 42604962 |
| Exposure to SARS-CoV-2 | 187.15 | 69.58 | 33 | 2967 | 565 | 42617770 |
| Product distribution issue | 120.55 | 69.58 | 28 | 2972 | 1966 | 42616369 |
| COVID-19 | 78.58 | 69.58 | 65 | 2935 | 115883 | 42502452 |
| Catheter site pain | 77.39 | 69.58 | 19 | 2981 | 1688 | 42616647 |
| 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 |
|---|---|---|---|---|---|---|
| No adverse event | 2140.47 | 75.86 | 460 | 2197 | 73379 | 110513263 |
| Product dose omission issue | 2012.55 | 75.86 | 583 | 2074 | 321199 | 110265443 |
| Intentional dose omission | 1261.91 | 75.86 | 240 | 2417 | 19840 | 110566802 |
| Poor venous access | 559.05 | 75.86 | 127 | 2530 | 24395 | 110562247 |
| Device issue | 536.18 | 75.86 | 131 | 2526 | 34404 | 110552238 |
| Exposure to SARS-CoV-2 | 152.00 | 75.86 | 30 | 2627 | 2889 | 110583753 |
| Product distribution issue | 123.23 | 75.86 | 27 | 2630 | 4319 | 110582323 |
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| Source | Code | Description |
|---|---|---|
| ATC | M09AX06 | 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 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 | Patent number | Patent expiration date | Patent use |
|---|---|---|---|---|---|---|---|---|---|---|
| 100MG/2ML (50MG/ML) | EXONDYS 51 | SAREPTA THERAPS INC | N206488 | Sept. 19, 2016 | RX | SOLUTION | INTRAVENOUS | RE48468 | Oct. 27, 2028 | TREATMENT OF DMD IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING |
| 500MG/10ML (50MG/ML) | EXONDYS 51 | SAREPTA THERAPS INC | N206488 | Sept. 19, 2016 | RX | SOLUTION | INTRAVENOUS | RE48468 | Oct. 27, 2028 | TREATMENT OF DMD IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING |
| 100MG/2ML (50MG/ML) | EXONDYS 51 | SAREPTA THERAPS INC | N206488 | Sept. 19, 2016 | RX | SOLUTION | INTRAVENOUS | 10337003 | March 14, 2034 | TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING |
| 100MG/2ML (50MG/ML) | EXONDYS 51 | SAREPTA THERAPS INC | N206488 | Sept. 19, 2016 | RX | SOLUTION | INTRAVENOUS | 10364431 | March 14, 2034 | RESTORING AN MRNA READING FRAME TO INDUCE DYSTROPHIN PROTEIN PRODUCTION IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING |
| 100MG/2ML (50MG/ML) | EXONDYS 51 | SAREPTA THERAPS INC | N206488 | Sept. 19, 2016 | RX | SOLUTION | INTRAVENOUS | 10364431 | March 14, 2034 | TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING |
| 100MG/2ML (50MG/ML) | EXONDYS 51 | SAREPTA THERAPS INC | N206488 | Sept. 19, 2016 | RX | SOLUTION | INTRAVENOUS | 9506058 | March 14, 2034 | RESTORING AN MRNA READING FRAME TO INDUCE DYSTROPHIN PROTEIN PRODUCTION IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING |
| 100MG/2ML (50MG/ML) | EXONDYS 51 | SAREPTA THERAPS INC | N206488 | Sept. 19, 2016 | RX | SOLUTION | INTRAVENOUS | 9506058 | March 14, 2034 | TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING |
| 500MG/10ML (50MG/ML) | EXONDYS 51 | SAREPTA THERAPS INC | N206488 | Sept. 19, 2016 | RX | SOLUTION | INTRAVENOUS | 10337003 | March 14, 2034 | TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING |
| 500MG/10ML (50MG/ML) | EXONDYS 51 | SAREPTA THERAPS INC | N206488 | Sept. 19, 2016 | RX | SOLUTION | INTRAVENOUS | 10364431 | March 14, 2034 | RESTORING AN MRNA READING FRAME TO INDUCE DYSTROPHIN PROTEIN PRODUCTION IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING |
| 500MG/10ML (50MG/ML) | EXONDYS 51 | SAREPTA THERAPS INC | N206488 | Sept. 19, 2016 | RX | SOLUTION | INTRAVENOUS | 10364431 | March 14, 2034 | TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING |
| 500MG/10ML (50MG/ML) | EXONDYS 51 | SAREPTA THERAPS INC | N206488 | Sept. 19, 2016 | RX | SOLUTION | INTRAVENOUS | 9506058 | March 14, 2034 | RESTORING AN MRNA READING FRAME TO INDUCE DYSTROPHIN PROTEIN PRODUCTION IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING |
| 500MG/10ML (50MG/ML) | EXONDYS 51 | SAREPTA THERAPS INC | N206488 | Sept. 19, 2016 | RX | SOLUTION | INTRAVENOUS | 9506058 | March 14, 2034 | TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY IN PATIENTS HAVING A MUTATION OF THE DMD GENE THAT IS AMENABLE TO EXON 51 SKIPPING |
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| Target | Class | Pharos | UniProt | Action | Type | Activity value (-log[M]) | Mechanism action | Bioact source | MoA source |
|---|---|---|---|---|---|---|---|---|---|
| exon 51 of dystrophin pre-mRNA | RNA | BINDING AGENT | UNKNOWN | DRUG LABEL |
| ID | Source |
|---|---|
| AIW6036FAS | UNII |
| 4036083 | VANDF |
| C4283710 | UMLSCUI |
| CHEBI:753271 | CHEBI |
| CHEMBL2108278 | ChEMBL_ID |
| D09900 | KEGG_DRUG |
| DB06014 | DRUGBANK_ID |
| 9339 | INN_ID |
| C000611335 | MESH_SUPPLEMENTAL_RECORD_UI |
| 7534 | IUPHAR_LIGAND_ID |
| 724033001 | SNOMEDCT_US |
| 763585001 | SNOMEDCT_US |
| 244179 | MMSL |
| 31970 | MMSL |
| d08476 | MMSL |
| 016988 | NDDF |
| 1810569 | RXNORM |
| Product | Category | Ingredients | NDC | Form | Quantity | Route | Marketing | Label |
|---|---|---|---|---|---|---|---|---|
| Exondys 51 | HUMAN PRESCRIPTION DRUG LABEL | 1 | 60923-284 | INJECTION | 50 mg | INTRAVENOUS | NDA | 27 sections |
| Exondys 51 | HUMAN PRESCRIPTION DRUG LABEL | 1 | 60923-363 | INJECTION | 50 mg | INTRAVENOUS | NDA | 27 sections |