risdiplam 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | PK/Tox | Related Drugs | BioActivity |

Stem definitionDrug idCAS RN
SMN2 gene splicing modulators (small molecules) 5407 1825352-65-5

Description:

MoleculeDescription

Molfile Inchi Smiles

Synonyms:

  • risdiplam
  • evrysdi
  • RG7916
  • RO7034067
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.
  • Molecular weight: 401.47
  • Formula: C22H23N7O
  • CLOGP: 2.42
  • LIPINSKI: 0
  • HAC: 8
  • HDO: 1
  • TPSA: 78.13
  • ALOGS: -3.62
  • ROTB: 2
  • Status: ONP
    • Legend:
    OFP - off patent
    OFM - off market
    ONP - on patent

Drug dosage:

DoseUnitRoute
5 mg O

ADMET properties:

PropertyValueReference
BDDCS (Biopharmaceutical Drug Disposition Classification System) 2 Bocci G, Oprea TI, Benet LZ

Predicted pharmacokinetic/toxicological properties (PK-AZ):

PropertyDescriptionValueUnitConfidenceSimilarity
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:

DateAgencyCompanyOrphan
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 termLikelihood ratioLikelihood ratio thresholdPatients taking drug having adverse eventPatients taking drug not having adverse eventPatients not taking drug having adverse eventPatients 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 termLikelihood ratioLikelihood ratio thresholdPatients taking drug having adverse eventPatients taking drug not having adverse eventPatients not taking drug having adverse eventPatients 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 termLikelihood ratioLikelihood ratio thresholdPatients taking drug having adverse eventPatients taking drug not having adverse eventPatients not taking drug having adverse eventPatients 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:

SourceCodeDescription
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
aceneuramic acidatalurencasimerseneteplirsengivinostatgolodirsenhyaluronic acidnusinersenonasemnogene abeparvovecpalovaroteneviltolarsen

Drug Use | Suggest Off label Use Form| |View source of the data|

DiseaseRelationSNOMED_IDDOID
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 strengthTrade nameApplicantApplication numberApproval dateTypeDose formRoutePatent numberPatent expiration datePatent 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 strengthTrade nameApplicantApplication numberApproval dateTypeDose formRouteExclusivity dateDescription
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:

IDSource
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:

ProductCategoryIngredientsNDCFormQuantityRouteMarketingLabel
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