Spinraza
E24879
Spinraza is a prescription medication used to treat spinal muscular atrophy (SMA) by modifying SMN2 gene expression to increase production of survival motor neuron (SMN) protein.
Aliases (2)
- nusinersen ×4
- SMN2 ×1
Statements (49)
| Predicate | Object |
|---|---|
| instanceOf |
antisense oligonucleotide
→
biologic medicinal product → medication → orphan drug → prescription drug → |
| approvedBy |
European Medicines Agency
→
U.S. Food and Drug Administration → |
| developedBy |
Biogen
→
Ionis Pharmaceuticals → |
| hasActiveIngredient |
nusinersen
→
|
| hasAdministrationSite |
cerebrospinal fluid
→
|
| hasATCCode |
M09AX07
→
|
| hasBrandOwner |
Biogen
→
|
| hasCommonAdverseEffect |
back pain
→
constipation → headache → nausea → respiratory infection → |
| hasDosageForm |
solution for injection
→
|
| hasGenericName |
nusinersen
→
|
| hasInternationalNonproprietaryName |
nusinersen
→
|
| hasLoadingDoseRegimen |
four loading doses over approximately two months
→
|
| hasMaintenanceDoseRegimen |
one dose every four months
→
|
| hasPregnancyCategory |
not assigned; use only if potential benefit justifies potential risk
→
|
| hasSeriousAdverseEffect |
coagulation abnormalities
→
renal toxicity → thrombocytopenia → |
| hasTherapeuticArea |
neurology
→
rare diseases → |
| increasesProductionOf |
SMN protein
→
survival motor neuron protein → |
| indicatedFor |
adult patients with spinal muscular atrophy
→
pediatric patients with spinal muscular atrophy → |
| isAdministeredBy |
healthcare professional
→
|
| isFirstDrugApprovedFor |
spinal muscular atrophy
→
|
| isUsedAs |
disease-modifying therapy for spinal muscular atrophy
→
|
| legalStatusInEuropeanUnion |
prescription only
→
|
| legalStatusInUnitedStates |
prescription only
→
|
| mechanismOfAction |
antisense oligonucleotide that modulates SMN2 pre-mRNA splicing
→
|
| modifiesGeneExpression |
SMN2
→
|
| regulatoryApprovalYear |
2016
→
|
| requiresMonitoringOf |
coagulation parameters
→
platelet count → renal function → |
| routeOfAdministration |
intrathecal
→
lumbar puncture → |
| targetsGene |
SMN2
→
|
| treats |
5q spinal muscular atrophy
→
spinal muscular atrophy → |
Referenced by (9)
| Subject (surface form when different) | Predicate |
|---|---|
|
Ionis Pharmaceuticals
→
|
coDeveloped |
|
Spinraza
("nusinersen")
→
|
hasActiveIngredient |
|
Spinraza
("nusinersen")
→
|
hasGenericName |
|
Spinraza
("nusinersen")
→
|
hasInternationalNonproprietaryName |
|
Biogen
→
|
hasNotableDrug |
|
Biogen
→
|
hasProduct |
|
Ionis Pharmaceuticals
→
|
notableProduct |
|
Spinraza
("SMN2")
→
|
targetsGene |
|
spinal muscular atrophy
("nusinersen")
→
|
treatedBy |