Evrysdi(risdiplam)
Evrysdi (risdiplam) is a small molecule pharmaceutical. Risdiplam was first approved as Evrysdi on 2020-08-07. It has been approved in Europe to treat spinal muscular atrophy.
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Commercial
Therapeutic Areas
Therapeutic Area | MeSH |
|---|---|
| nervous system diseases | D009422 |
Trade Name
FDA
EMA
Evrysdi
Labels
FDA
EMA
Brand Name | Status | Last Update |
|---|---|---|
| evrysdi | New Drug Application | 2024-09-26 |
Indications
FDA
EMA
No data
Agency Specific
FDA
EMA
Expiration | Code | ||
|---|---|---|---|
RISDIPLAM, EVRYSDI, GENENTECH INC | |||
| 2029-05-27 | ODE-400 | ||
| 2027-08-07 | ODE-334 | ||
| 2026-10-03 | M-270 | ||
| 2025-08-07 | NCE | ||
| 2025-05-27 | NPP | ||
HCPCS
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Clinical
Clinical Trials
27 clinical trials
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Indications Phases 4
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Muscular atrophy | D009133 | HP_0003202 | — | 4 | 6 | 4 | 4 | 7 | 24 |
| Spinal muscular atrophy | D009134 | EFO_0003823 | G12.1 | 4 | 6 | 4 | 4 | 7 | 24 |
| Atrophy | D001284 | — | — | — | 6 | 4 | 4 | 6 | 19 |
Indications Phases 3
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Spinal muscular atrophies of childhood | D014897 | Orphanet_83419 | G12.1 | — | — | 1 | — | — | 1 |
| Neuromuscular diseases | D009468 | EFO_1001902 | G70.9 | — | — | 1 | — | — | 1 |
| Neuromuscular manifestations | D020879 | — | — | — | — | 1 | — | — | 1 |
Indications Phases 2
No data
Indications Phases 1
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Healthy volunteers/patients | — | — | — | 2 | — | — | — | — | 2 |
Indications Without Phase
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Fatigue | D005221 | HP_0012378 | R53.83 | — | — | — | — | 1 | 1 |
| Nervous system diseases | D009422 | — | G00-G99 | — | — | — | — | 1 | 1 |
| Amyotrophic lateral sclerosis | D000690 | HP_0007354 | G12.21 | — | — | — | — | 1 | 1 |
| Motor neuron disease | D016472 | EFO_0003782 | G12.2 | — | — | — | — | 1 | 1 |
Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
| Drug common name | Risdiplam |
| INN | risdiplam |
| Description | Risdiplam, sold under the brand name Evrysdi, is a medication used to treat spinal muscular atrophy (SMA) and the first oral medication approved to treat this disease.
|
| Classification | Small molecule |
| Drug class | survival motor neurin SMN1 and SMN2 splicing modulators |
| Image (chem structure or protein) |  |
| Structure (InChI/SMILES or Protein Sequence) | Cc1cn2nc(-c3cc(=O)n4cc(N5CCNC6(CC6)C5)ccc4n3)cc(C)c2n1 |
Identifiers
| PDB | — |
| CAS-ID | 1825352-65-5 |
| RxCUI | — |
| ChEMBL ID | CHEMBL4297528 |
| ChEBI ID | — |
| PubChem CID | 118513932 |
| DrugBank | DB15305 |
| UNII ID | 76RS4S2ET1 (ChemIDplus, GSRS) |
Target
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Variants
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Financial
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Evrysdi – Roche
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Tabular view
Estimated US medical usage
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Trends
PubMed Central
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Additional graphs summarizing 875 documents
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Safety
Black-box Warning
No Black-box warning
Adverse Events
Top Adverse Reactions
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2,132 adverse events reported
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