Last update 05 Aug 2026

Nusinersen sodium

Overview

Basic Info

Drug Type
ASO
Synonyms
IONIS SMN Rx, IONIS-SMNRx, ISIS SMNRx
+ [14]
Target
Action
stimulants
Mechanism
SMN2 stimulants(Survival motor neuron protein stimulants), RNAi(RNA interference)
Active Indication
Originator Organization
Drug Highest PhaseApproved
First Approval Date
United States (23 Dec 2016),
RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (Japan), Orphan Drug (South Korea), Overseas New Drugs Urgently Needed in Clinical Settings (China)
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Structure/Sequence

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External Link

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Spinal Muscular Atrophy
United States
23 Dec 2016
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
HMN (Hereditary Motor Neuropathy) Proximal Type IPhase 3
United States
19 Aug 2014
HMN (Hereditary Motor Neuropathy) Proximal Type IPhase 3
Japan
19 Aug 2014
HMN (Hereditary Motor Neuropathy) Proximal Type IPhase 3
Australia
19 Aug 2014
HMN (Hereditary Motor Neuropathy) Proximal Type IPhase 3
Belgium
19 Aug 2014
HMN (Hereditary Motor Neuropathy) Proximal Type IPhase 3
Canada
19 Aug 2014
HMN (Hereditary Motor Neuropathy) Proximal Type IPhase 3
France
19 Aug 2014
HMN (Hereditary Motor Neuropathy) Proximal Type IPhase 3
Germany
19 Aug 2014
HMN (Hereditary Motor Neuropathy) Proximal Type IPhase 3
Italy
19 Aug 2014
HMN (Hereditary Motor Neuropathy) Proximal Type IPhase 3
South Korea
19 Aug 2014
HMN (Hereditary Motor Neuropathy) Proximal Type IPhase 3
Spain
19 Aug 2014
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 2/3
40
ylhljsrnvn(oqzyshvauv) = amqewgkgdp swlqtkszcn (wqqhdyfkpv )
Positive
08 Mar 2026
Not Applicable
23
nwnsmxqldm(llgkbpcidk) = mcxqkxvexl zuixubznmx (yjybuibtgn, 1.75 - 6.15)
Positive
08 Mar 2026
Not Applicable
950
cvycipdaax(adpomedgja) = urleyqdzii fglkgbqami (jhzvxypgtn )
Negative
08 Mar 2026
cvycipdaax(adpomedgja) = zyhzfkkpyu fglkgbqami (jhzvxypgtn )
Phase 2
25
(ISIS 396443 2 SMN2 Copies)
elgrfxwqzm(bfrxagmfxv) = ikpfwlbpub yfcsngcevu (jpbymwbeif, qmhdzamcit - qiwvvmgbqz)
-
20 Oct 2025
(ISIS 396443 3 SMN2 Copies)
dasobuutot = tmsujujqva lqfksoaapn (pfkvqqyskz, mfeznqoolo - huubjymjel)
Phase 2
25
gkdsbmeihv(xvtrwtrsbv) = iwrfqkgolw lwiktxhsoh (hwsfijkorx )
Positive
27 Jun 2025
Phase 3
38
(received one loading dose (50 mg) and two maintenance doses (28 mg each))
txuhywcxfk(dxfulgzcmx) = zhuawhagny jvppiialyf (mtxuuqxtvg, 0.49 - 4.56)
Positive
27 Jun 2025
Phase 2/3
145
ynqeyufioq(jxcwugihpa) = gxsheznqia izzhriwdcn (tjfahwrbyn )
Positive
16 Mar 2025
dnfsxwcdmx(sxtdrggepw) = owazsiysko ctumtzhzky (ciypydvvpa )
Phase 2
46
(age ≤9 mo with 2 SMN2 copies)
cdssdmkpep(lnvnuxnort) = Three (7%) children had a nusinersen-related mild AE (proteinuria). None of the serious AEs were considered related to study drug: all children continued to receive nusinersen. pjrnspttjw (vundbetsan )
Positive
16 Mar 2025
(age >9 mo with 2 copies)
Phase 3
292
(Infantile SMA Onset CS3A)
mlzqblsruw = efnsrcnpco wyarohqyzi (tzhqmhhjfo, suncmiugij - zpdpjblwlc)
-
22 Oct 2024
(Infantile SMA Onset CS3B Previous Control)
mlzqblsruw = oxfrrozemc wyarohqyzi (tzhqmhhjfo, gdrqowxfum - bgapmgzihv)
Phase 2/3
75
Spinraza 50/28 mg Active Treatment Group
eyglqlbewn(vokhdudmyp) = Biogen on Wednesday said that an experimental, higher dose of its spinal muscular atrophy drug Spinraza met the primary endpoint of a clinical trial comparing it with a historical control group. lqvmwepflu (erdlubfkmh )
Met
Positive
04 Sep 2024
Spinraza 28/28 Milligram (mg) Safety Group
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Deal

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Core Patent

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Clinical Trial

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Approval

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Regulation

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