risdiplam

E133682

Risdiplam is an oral small-molecule drug used to treat spinal muscular atrophy by increasing production of functional survival motor neuron (SMN) protein.

All labels observed (1)

Label Occurrences
risdiplam canonical 2

How this entity was disambiguated

Statements (49)

Predicate Object
instanceOf disease-modifying therapy for spinal muscular atrophy
orally administered medication
small-molecule drug
splicing modifier
actsOnGene SMN2
administeredOnce daily
administrationPopulation adults
children
infants
administrationWithFood can be taken with or without food
affectsProtein survival motor neuron protein
surface form: SMN protein
approvedBy European Medicines Agency
Food and Drug Administration
surface form: U.S. Food and Drug Administration
clinicalTrialProgram FIREFISH
JEWELFISH
RAINBOWFISH
SUNFISH
commonAdverseEvent diarrhea
fever
rash
upper respiratory tract infection
contraindication known hypersensitivity to risdiplam or its excipients
developedBy PTC Therapeutics
Roche
developedInCollaborationWith SMA Foundation
dosageForm oral solution
eliminationRoute mainly via feces
hasATCCode M09AX10
hasBrandName Evrysdi
hasMolecularTarget SMN2
surface form: SMN2 pre-mRNA
improvesClinicalOutcome motor function in patients with spinal muscular atrophy
increasesProductionOf functional SMN protein
indicatedFor 5q-associated spinal muscular atrophy
indication treatment of spinal muscular atrophy in adult patients
treatment of spinal muscular atrophy in pediatric patients
mechanismOfAction modulation of SMN2 pre-mRNA splicing to promote exon 7 inclusion
metabolism primarily hepatic
pharmacologicalClass SMN2 splicing modifier
survival motor neuron protein upregulator
pregnancyCategory may cause fetal harm based on animal data
regulatoryDesignation orphan drug for spinal muscular atrophy
regulatoryStatus prescription-only medicine
requiresMonitoringOf liver function
ophthalmologic status in some patients
routeOfAdministration oral
storageCondition refrigeration before reconstitution (for some formulations)
targetsBiologicalProcess pre-mRNA splicing of SMN2
therapeuticEffect increase in SMN protein levels in blood and tissues
treats spinal muscular atrophy

How these facts were elicited

Referenced by (2)

Full triples — surface form annotated when it differs from this entity's canonical label.

SMN2 targetOfDrug risdiplam