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risdiplam 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | PK/Tox | Related Drugs | BioActivity |
| Stem definition | Drug id | CAS RN |
|---|---|---|
| SMN2 gene splicing modulators (small molecules) | 5407 | 1825352-65-5 |
Description:
| Molecule | Description |
|---|---|
|
Synonyms:
|
Risdiplam is a survival of motor neuron 2 (SMN2) splicing modifier designed to treat patients with spinal muscular atrophy (SMA) caused by mutations in chromosome 5q that lead to SMN protein deficiency. Using in vitro assays and studies in transgenic animal models of SMA, risdiplam was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein in the brain.
Legend: OFP - off patent OFM - off market ONP - on patent |
Drug dosage:
| Dose | Unit | Route |
|---|---|---|
| 5 | mg | O |
ADMET properties:
| Property | Value | Reference |
|---|---|---|
| BDDCS (Biopharmaceutical Drug Disposition Classification System) | 2 | Bocci G, Oprea TI, Benet LZ |
Predicted pharmacokinetic/toxicological properties (PK-AZ):
| Property | Description | Value | Unit | Confidence | Similarity |
|---|---|---|---|---|---|
| kinase-safety-class | ACVR2B,AXL,BRAF,CAMK2D,CDK9,EIF2AK3,ERBB2,FLT3,GRK2,IRAK1,ITK,KIT,MAP3K21,MAP4K1,MAPK7,NTRK1,PDK1,PDK2,PDK4,PIK3CA,PIK3CB,PIK3CD,PRKDC,SIK2 | 24 | |||
| kinase-selectivity-class | PDK1 | 1 | |||
| pfas-class | PFAS structural alert (1 = True, 0 = False) | 0 |
Approvals:
| Date | Agency | Company | Orphan |
|---|---|---|---|
| Aug. 7, 2020 | FDA | GENENTECH INC | |
| March 26, 2021 | EMA | Roche Registration GmbH | |
| June 23, 2021 | PMDA | CHUGAI PHARMACEUTICAL Co., Ltd. |
FDA Adverse Event Reporting System (Female)
| 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 | 139.41 | 38.35 | 48 | 1292 | 67934 | 90559173 |
| Respiratory tract infection | 73.75 | 38.35 | 29 | 1311 | 59049 | 90568058 |
FDA Adverse Event Reporting System (Male)
| 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 | 95.23 | 39.60 | 34 | 832 | 38987 | 42581482 |
| Respiratory tract infection | 83.80 | 39.60 | 28 | 838 | 26294 | 42594175 |
| Death | 39.83 | 39.60 | 48 | 818 | 471137 | 42149332 |
FDA Adverse Event Reporting System (Geriatric)
| 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 |
|---|---|---|---|---|---|---|
| Respiratory tract infection | 149.08 | 37.34 | 52 | 1690 | 71565 | 110515992 |
| No adverse event | 117.55 | 37.34 | 44 | 1698 | 73795 | 110513762 |
| Diarrhoea | 57.11 | 37.34 | 79 | 1663 | 1139426 | 109448131 |
| Scoliosis | 50.91 | 37.34 | 14 | 1728 | 8719 | 110578838 |
| Pneumonia | 45.60 | 37.34 | 62 | 1680 | 876652 | 109710905 |
| COVID-19 | 39.77 | 37.34 | 32 | 1710 | 245028 | 110342529 |
| Aphthous ulcer | 38.73 | 37.34 | 14 | 1728 | 21122 | 110566435 |
FDA Adverse Event Reporting System (Pediatric)
None
Pharmacologic Action:
| Source | Code | Description |
|---|---|---|
| ATC | M09AX10 | 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 | D009465 | Neuromuscular Agents |
| MeSH PA | D018373 | Peripheral Nervous System Agents |
| FDA PE | N0000009533 | Increased Protein Synthesis |
| FDA MoA | N0000191423 | Multidrug and Toxin Extrusion Transporter 1 Inhibitors |
| FDA MoA | N0000193932 | Multidrug and Toxin Extrusion Transporter 2 K Inhibitors |
| FDA EPC | N0000194059 | Survival of Motor Neuron 2 Splicing Modifier |
| FDA MoA | N0000194060 | Survival of Motor Neuron 2 Splicing Modifiers |
Related Drugs by ATC class:
M09AX Other drugs for disorders of the musculo-skeletal system 11 drugs
Drug Use | Suggest Off label Use Form| |View source of the data|
| Disease | Relation | SNOMED_ID | DOID |
|---|---|---|---|
| Spinal muscular atrophy | indication | 5262007 | DOID:12377 |
🐶 Veterinary Drug Use
None
🐶 Veterinary products
None
Acid dissociation constants calculated using MoKa v3.0.0
None
Orange Book patent data (new drug applications)
| Formulation strength | Trade name | Applicant | Application number | Approval date | Type | Dose form | Route | Patent number | Patent expiration date | Patent use |
|---|---|---|---|---|---|---|---|---|---|---|
| 0.75MG/ML | EVRYSDI | GENENTECH INC | N213535 | Aug. 7, 2020 | RX | FOR SOLUTION | ORAL | 9969754 | May 11, 2035 | TREATMENT OF SPINAL MUSCULAR ATROPHY |
| 5MG | EVRYSDI | GENENTECH INC | N219285 | Feb. 11, 2025 | RX | TABLET | ORAL | 9969754 | May 11, 2035 | TREATMENT OF SPINAL MUSCULAR ATROPHY |
| 0.75MG/ML | EVRYSDI | GENENTECH INC | N213535 | Aug. 7, 2020 | RX | FOR SOLUTION | ORAL | 11827646 | Jan. 25, 2036 | TREATMENT OF SPINAL MUSCULAR ATROPHY |
| 5MG | EVRYSDI | GENENTECH INC | N219285 | Feb. 11, 2025 | RX | TABLET | ORAL | 11827646 | Jan. 25, 2036 | TREATMENT OF SPINAL MUSCULAR ATROPHY |
| 0.75MG/ML | EVRYSDI | GENENTECH INC | N213535 | Aug. 7, 2020 | RX | FOR SOLUTION | ORAL | 12350273 | Oct. 1, 2038 | TREATMENT OF SPINAL MUSCULAR ATROPHY |
| 5MG | EVRYSDI | GENENTECH INC | N219285 | Feb. 11, 2025 | RX | TABLET | ORAL | 12350273 | Oct. 1, 2038 | TREATMENT OF SPINAL MUSCULAR ATROPHY |
| 0.75MG/ML | EVRYSDI | GENENTECH INC | N213535 | Aug. 7, 2020 | RX | FOR SOLUTION | ORAL | 11534444 | Oct. 4, 2038 | TREATMENT OF SPINAL MUSCULAR ATROPHY |
| 5MG | EVRYSDI | GENENTECH INC | N219285 | Feb. 11, 2025 | RX | TABLET | ORAL | 11534444 | Oct. 4, 2038 | TREATMENT OF SPINAL MUSCULAR ATROPHY |
Orange Book exclusivity data (new drug applications)
| Formulation strength | Trade name | Applicant | Application number | Approval date | Type | Dose form | Route | Exclusivity date | Description |
|---|---|---|---|---|---|---|---|---|---|
| 0.75MG/ML | EVRYSDI | GENENTECH INC | N213535 | Aug. 7, 2020 | RX | FOR SOLUTION | ORAL | Oct. 3, 2026 | INFORMATION ADDED TO CLINICAL PHARMACOLOGY SECTION |
| 5MG | EVRYSDI | GENENTECH INC | N219285 | Feb. 11, 2025 | RX | TABLET | ORAL | Oct. 3, 2026 | INFORMATION ADDED TO CLINICAL PHARMACOLOGY SECTION |
| 0.75MG/ML | EVRYSDI | GENENTECH INC | N213535 | Aug. 7, 2020 | RX | FOR SOLUTION | ORAL | Aug. 7, 2027 | TREATMENT OF SPINAL MUSCULAR ATROPHY (SMA) IN PATIENTS 2 MONTHS OF AGE AND OLDER |
| 0.75MG/ML | EVRYSDI | GENENTECH INC | N213535 | Aug. 7, 2020 | RX | FOR SOLUTION | ORAL | May 27, 2029 | TREATMENT OF SPINAL MUSCULAR ATROPHY (SMA) IN PEDIATRIC PATIENTS BETWEEN BIRTH AND 2 MONTHS OF AGE |
Bioactivity Summary:
None
External reference:
| ID | Source |
|---|---|
| D11406 | KEGG_DRUG |
| 76RS4S2ET1 | UNII |
| C5139676 | UMLSCUI |
| CHEBI:233561 | CHEBI |
| CHEMBL4297528 | ChEMBL_ID |
| 118513932 | PUBCHEM_CID |
| DB15305 | DRUGBANK_ID |
| 10614 | INN_ID |
| C000629884 | MESH_SUPPLEMENTAL_RECORD_UI |
| 11170 | IUPHAR_LIGAND_ID |
| 896813005 | SNOMEDCT_US |
| 896965008 | SNOMEDCT_US |
| 4039628 | VANDF |
| 337348 | MMSL |
| 38721 | MMSL |
| d09615 | MMSL |
| 018470 | NDDF |
| 2390935 | RXNORM |
Pharmaceutical products:
| Product | Category | Ingredients | NDC | Form | Quantity | Route | Marketing | Label |
|---|---|---|---|---|---|---|---|---|
| EVRYSDI | HUMAN PRESCRIPTION DRUG LABEL | 1 | 50242-175 | POWDER, FOR SOLUTION | 0.75 mg | ORAL | NDA | 30 sections |
| EVRYSDI | HUMAN PRESCRIPTION DRUG LABEL | 1 | 50242-202 | TABLET | 5 mg | ORAL | NDA | 30 sections |