|
gptkbp:instanceOf
|
gptkb:drug
|
|
gptkbp:approvalYear
|
2016
|
|
gptkbp:ATCCode
|
gptkb:M09AX07
|
|
gptkbp:bioavailability
|
not applicable (intrathecal)
|
|
gptkbp:blackBoxWarning
|
no
|
|
gptkbp:brand
|
gptkb:Spinraza
|
|
gptkbp:CASNumber
|
1258984-36-9
|
|
gptkbp:contraindication
|
hypersensitivity to nusinersen
|
|
gptkbp:cost
|
high (>$100,000 per dose)
|
|
gptkbp:developer
|
gptkb:Biogen
gptkb:Ionis_Pharmaceuticals
|
|
gptkbp:drugClass
|
antisense oligonucleotide
|
|
gptkbp:halfLife
|
135-177 days (CSF)
|
|
gptkbp:hasAdministrationFrequency
|
every 4 months after loading doses
|
|
gptkbp:hasCountryOfApproval
|
gptkb:European_Union
gptkb:United_States
|
|
gptkbp:hasDosageForm
|
solution for injection
|
|
gptkbp:hasFirstInClass
|
yes
|
|
gptkbp:hasMolecularFormula
|
C234H362N61O128P17S17
|
|
gptkbp:hasOrphanDrugStatus
|
yes
|
|
gptkbp:hasPharmacokinetics
|
not systemically distributed
|
|
gptkbp:hasSMILES
|
CC1=CC=CC=C1C(=O)O... (truncated)
|
|
gptkbp:hasStorageCondition
|
refrigerated
|
|
gptkbp:hasUNII
|
6F4V4A6EZE
|
|
https://www.w3.org/2000/01/rdf-schema#label
|
DB11162
|
|
gptkbp:indication
|
spinal muscular atrophy
|
|
gptkbp:legalStatus
|
prescription only
patented
|
|
gptkbp:mechanismOfAction
|
antisense oligonucleotide that modulates splicing of SMN2 pre-mRNA
|
|
gptkbp:molecularWeight
|
7500.0
|
|
gptkbp:name
|
Nusinersen
|
|
gptkbp:pregnancyCategory
|
not assigned
|
|
gptkbp:PubChem_CID
|
gptkb:DB11162
gptkb:CHEMBL3989989
D10572
71711961
|
|
gptkbp:routeOfAdministration
|
intrathecal
|
|
gptkbp:sideEffect
|
respiratory infection
constipation
headache
back pain
|
|
gptkbp:status
|
FDA approved
EMA approved
|
|
gptkbp:synonym
|
ISIS 396443
ISIS-SMNrx
SPINRAZA
|
|
gptkbp:target
|
gptkb:SMN2_gene
|
|
gptkbp:therapeuticArea
|
neurology
|
|
gptkbp:bfsParent
|
gptkb:sodium_butyrate
|
|
gptkbp:bfsLayer
|
8
|