Explore drug properties, targets, and indications with answers grounded in DrugCentral.
Powered by DrugCentral + retrieval-augmented AIAsk in your own words. Each question is answered independently.
Select a question to fill the editor, then ask it or make it your own.
Your answer will appear here. Open linked drug cards in a new tab to explore the source details.
Ask DrugCentral helps you explore drug properties, biological targets, and indications using natural language. Sample questions offer starting points, and linked drug cards let you check the underlying records.
Each request is independent; previous questions are not sent as conversation history. You can edit a sample before submitting, retry a failed request, or switch back to normal search. Drug links open in a new tab so your question and answer stay available.
Answers may contain mistakes or omit information. Verify details against DrugCentral records and original sources.
AI can make mistakes. Verify answers against the original sources before relying on them.
| Stem definition | Drug id | CAS RN |
|---|---|---|
| antisense oligonucleotides | 5182 | 1173755-55-9 |
| Molecule | Description |
|---|---|
|
Synonyms:
|
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.
|
None
None
None
| Date | Agency | Company | Orphan |
|---|---|---|---|
| Sept. 19, 2016 | FDA | SAREPTA THERAPEUTICS INC |
None
| 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 |
None
| 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 |
None
None
None
| 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 |
None
| 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 |