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risdiplam   Click here for help

GtoPdb Ligand ID: 11170

Synonyms: compound 1 [PMID: 30044619] | Evrysdi® | RG-7916 | RG7916 | RO-7034067 | RO7034067
Approved drug
risdiplam is an approved drug (FDA (2020), EMA (2021))
Compound class: Synthetic organic
Comment: Risdiplam is a first-in-class, orally bioavailable splicing modifier [3]. Like the splice switching oligonucleotide nusinersen, risdiplam is intended to improve the efficiency of SMN2 (HGNC ID 11118; Uniprot Q16637) gene transcription in patients with spinal muscular atrophy (SMA) [2]. Risdiplam binds directly to the SMN2 pre-mRNA to modulate its splicing pattern to produce a protein that compensates for the defective SMN1 gene.

risdiplam is commercially available from our sponsors

2D Structure
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2D Structure

An image of the ligand's 2D structure. For small molecules with SMILES these are drawn using the NCI/CADD Chemical Identifier Resolver. Click on the image to access the chemical structure search tool with the ligand pre-loaded in the structure editor. For other types of ligands, e.g. longer nucleotides and peptides, a manually drawn representation of the molecule may be provided.

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Physico-chemical Properties
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Physico-chemical Properties

Calculated molecular properties are available for small molecules and natural products (not peptides). Properties were generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/). All properties were selected to enable the prediction of the Lipinski Rule-of-Five profile or ‘druglikeness’ for each ligand. For more info on each category see the help pages.

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Hydrogen bond acceptors 5
Hydrogen bond donors 1
Rotatable bonds 2
Topological polar surface area 79.83
Molecular weight 401.2
XLogP 3.52
No. Lipinski's rules broken 0

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)
SMILES / InChI / InChIKey
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SMILES / InChI / InChIKey

SMILES (Simplified Molecular Input Line Entry Specification) A specification for unambiguously describing the structure of chemical molecules using short ASCII strings. Canonical SMILES specify a unique representation of the 2D structure without chiral or isotopic specifications. Isomeric SMILES include chiral specification and isotopes.

Standard InChI (IUPAC International Chemical Identifier) and InChIKey InChI is a non-proprietary, standard, textual identifier for chemical substances designed to facilitate linking of information and database searching. An InChIKey is a simplified version of a full InChI, designed for easier web searching.

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)

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Canonical SMILES Cc1cn2c(n1)c(C)cc(n2)c1cc(=O)n2c(n1)ccc(c2)N1CCNC2(C1)CC2
Isomeric SMILES Cc1cn2c(n1)c(C)cc(n2)c1cc(=O)n2c(n1)ccc(c2)N1CCNC2(C1)CC2
InChI InChI=1S/C22H23N7O/c1-14-9-18(26-29-11-15(2)24-21(14)29)17-10-20(30)28-12-16(3-4-19(28)25-17)27-8-7-23-22(13-27)5-6-22/h3-4,9-12,23H,5-8,13H2,1-2H3
InChI Key ASKZRYGFUPSJPN-UHFFFAOYSA-N

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)
Classification Click here for help
Compound class Synthetic organic
Approved drug? Yes. EU EMA (2021)  |  US FDA (2020)
International Nonproprietary Names Click here for help
INN number INN
10614 risdiplam
Synonyms Click here for help
compound 1 [PMID: 30044619] | Evrysdi® | RG-7916 | RG7916 | RO-7034067 | RO7034067
Database Links Click here for help
ChEMBL Ligand CHEMBL4297528
DrugBank Ligand DB15305
DrugCentral Ligand 5407
GtoPdb PubChem SID 434122230
PubChem CID 118513932
Search Google for chemical match using the InChIKey ASKZRYGFUPSJPN-UHFFFAOYSA-N
Search Google for chemicals with the same backbone ASKZRYGFUPSJPN
Search PubMed clinical trials risdiplam
Search PubMed titles risdiplam
Search PubMed titles/abstracts risdiplam
UniChem Compound Search for chemical match using the InChIKey ASKZRYGFUPSJPN-UHFFFAOYSA-N
UniChem Connectivity Search for chemical match using the InChIKey ASKZRYGFUPSJPN-UHFFFAOYSA-N

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Product supplier links are provided as a service to assist in identifying commercial suppliers of reagents that are mentioned on the IUPHAR/BPS Guide to PHARMACOLOGY database website, and do not imply their endorsement by NC-IUPHAR.

Links are provided in return for sponsorship, used to fund improvements to this database. The sponsorship account is managed and audited by the University of Edinburgh, a charitable body registered in Scotland, SC005336. If you are interested in sponsoring the database, please contact us.

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MedChemExpress
Risdiplam (links to external site)
Cat. No. HY-109101