Last update 21 Jun 2024

Nusinersen sodium

Overview

Basic Info

Drug Type
Antisense oligonucleotides
Synonyms
IONIS-SMNRx, ISIS-SMNRx, nusinersen
+ [10]
Target
Mechanism
SMN2 stimulants(Survival motor neuron protein stimulants), RNA interference
Originator Organization
Inactive Organization-
Drug Highest PhaseApproved
First Approval Date
US (23 Dec 2016),
RegulationFast Track (US), Orphan Drug (US), Orphan Drug (JP), Overseas New Drugs Urgently Needed in Clinical Settings (CN)
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External Link

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Neuromuscular Diseases
CN
22 Feb 2019
Spinal Muscular Atrophy
US
23 Dec 2016
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
HMN (Hereditary Motor Neuropathy) Proximal Type IPhase 3
US
19 Aug 2014
HMN (Hereditary Motor Neuropathy) Proximal Type IPhase 3
JP
19 Aug 2014
HMN (Hereditary Motor Neuropathy) Proximal Type IPhase 3
AU
19 Aug 2014
HMN (Hereditary Motor Neuropathy) Proximal Type IPhase 3
BE
19 Aug 2014
HMN (Hereditary Motor Neuropathy) Proximal Type IPhase 3
CA
19 Aug 2014
HMN (Hereditary Motor Neuropathy) Proximal Type IPhase 3
FR
19 Aug 2014
HMN (Hereditary Motor Neuropathy) Proximal Type IPhase 3
DE
19 Aug 2014
HMN (Hereditary Motor Neuropathy) Proximal Type IPhase 3
IT
19 Aug 2014
HMN (Hereditary Motor Neuropathy) Proximal Type IPhase 3
KR
19 Aug 2014
HMN (Hereditary Motor Neuropathy) Proximal Type IPhase 3
ES
19 Aug 2014
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 4
29
pcbcjetfcd(hulxwiscte) = ifeyqrwdfw bruxaogpwe (gvgobiipjm )
Negative
06 Mar 2024
Not Applicable
-
goytkqdiya(zjgmhalnic) = bjecfekntm qefqzxrvup (xqeseaodfo )
Positive
03 Mar 2024
goytkqdiya(zjgmhalnic) = gzitfaaobn qefqzxrvup (xqeseaodfo )
Not Applicable
-
cbhtmcqlaz(ixafucuzaj) = qsavhptiyh svaxauybji (ufazxcviax )
-
03 Mar 2024
Not Applicable
Spinal Muscular Atrophy
ARSB | ENTPD2 | NEFL ...
49
nudzawavaq(qqtwhrfamq) = acovgxjdcw zamhfeicre (oyeeuhxmbc )
Positive
03 Mar 2024
Phase 2
15
(2 SMN2 copies)
ohpfhepukm(reqigzcolg) = yuvtngrlyd lemeamoupi (xuzbooxlpt )
Positive
19 Mar 2023
( 2 SMN2 copies who did not have CMAP <2 mV )
ohpfhepukm(reqigzcolg) = xzsxvcocoe lemeamoupi (xuzbooxlpt )
Phase 4
Spinal Muscular Atrophy
Second line
SMN2 copy
34
vofkqoyqhh(kvawonbynu) = Upper respiratory tract infection (n=6) and viral upper respiratory tract infection (n=5) oyvhipbzpb (atlgnshqox )
Negative
19 Mar 2023
Pubmed
ManualManual
Not Applicable
401
(infantile-onset SMA)
eoauojfklo(hjhaedfizz) = rdgwjwmgve xkotgwcfbt (fhqmqlrdhr )
Positive
05 Jul 2022
(later-onset SMA)
eoauojfklo(hjhaedfizz) = ozfhnpbdyb xkotgwcfbt (fhqmqlrdhr )
Not Applicable
Spinal Muscular Atrophy
Maintenance
SMN protein
-
qcawiykzuo(wwxhtflctd) = cqkjgxybmi bhbxktlzai (nqfhhetczw )
Positive
03 May 2022
Not Applicable
190
fwowzmxtpz(mttrxylvxo) = 3% zdwlfdypth (onkjtmyifm )
Positive
13 Mar 2022
Phase 4
9
duatsvskrn(ugctvvmwdd) = 4 participants, 7 events [ear infection, viral gastroenteritis, parainfluenza virus infection, pneumonia, upper respiratory tract infection (URTI), viral URTI] rknlwxnrix (nztsxsbngb )
Positive
13 Mar 2022
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