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 8

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 SMN protein ⓘ
approvedBy European Medicines Agency ⓘ
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 pre-mRNA ⓘ
linked to: SMN2
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 (8)

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

SMN2 → targetOfDrug → risdiplam ⓘ
Evrysdi → hasActiveIngredient → risdiplam ⓘ
SUNFISH → evaluates → risdiplam ⓘ
JEWELFISH → evaluates → risdiplam ⓘ
JEWELFISH → hasOralFormulation → risdiplam ⓘ
RAINBOWFISH → evaluates → risdiplam ⓘ
RAINBOWFISH → hasIntervention → risdiplam ⓘ