Nusinersen
Also known as Spinraza, Nusinersen sodium
BIOGEN INC. BIIB · Spinal Muscular Atrophy; Muscular Atrophy, Spinal
Key figures
Next catalyst
None scheduled
Only dates with a source are listed
Phase
Approved
Not recorded
Linked trials
6
From ClinicalTrials.gov
Latest FDA action
Approved
NDA209531,
Recalls, shortages, patents and EU status at a glance
Key information
- Sponsor
- BIOGEN INC. BIIB Source: Company record for BIOGEN INC. (opens in a new tab)
- Indications
- Spinal Muscular Atrophy; Muscular Atrophy, Spinal Source: Conditions from ClinicalTrials.gov (opens in a new tab)
- Application
- NDA 209531 · Priority review Source: Drugs@FDA via openFDA (opens in a new tab) · Action letter for NDA 209531 (opens in a new tab)
- Designations
- None recorded
- Next catalyst
- None scheduled
Development timeline
- Preclinical Completed
- Phase 1 Completed
- Phase 2 Completed
- Phase 3 CompletedFirst trial start 19 Aug 20146 linked trials
- Filed Completed
- Approved Current stageFirst approval 23 Dec 2016
Overview
- Mechanism
- Not recorded
- Route
- Not recorded
- Modality
- Not recorded
- Specialties
- Not recorded
Overview text is written by HealthTrade editors.
Key dates
NusinersenPDUFAOccurred
BIOGEN INC.: PDUFA date in EX-99.1 (8-K) filed 30 October 2025
continued generic erosion of TECFIDERA in Europe Delivered progress across key late-stage pipeline programs during the third quarter •LEQEMBI IQLIK, the first and only FDA-approved anti-amyloid treatment to offer an at-home subcutaneous injection in a maintenance setting and launched in October 2025. FDA rolling submission underway for LEQEMBI IQLIK treatment initiation •Today announced that high dose nusinersen (SPINRAZA) in SMA was successfully resubmitted with the FDA with an updated PDUFA date of April 3, 2026 •Today announced both litifilimab Phase 3 studies for systemic lupus erythematosus fully enrolled with expected data readout for both studies now accelerated to H2 2026 •New analysis of dapirolizumab pegol Phase 3 data presented at American College of Rheumatology Convergence underscores the potential for a differentiated profile in systemic lupus erythematosus Advanced Biogen’s immunology strategy with new early-stage programs •IRAK4 degrader (BIIB142) for autoimmune disease, including lupus, initiated Phase 1 study in healthy volunteers •Added a preclinical C5aR1 antagonist through a licensing agreement with Vanqua Bio, adding a proven immune mechanism with the potential to address a broad range of inflammatory disorders Biogen updates full year 2025 guidance to reflect stronger underlying business outlook and investment for growth from business development transactions expected to close in the fourth quarter •Full year 2025 Non-GAAP diluted EPS expected to be between $14.50 and $15.00, including expected improved business impact of approximately $0.25 EPS, offset by an expected approximately ($1.25) EPS impact in the fourth quarter from acquired IPR&D expense •Increased expected full year 2025 total revenue to be approximately flat to increasing 1%, at constant currency, versus full year 2024, up from approximately flat previously 1 Biogen Inc.
213days ago
FDA designations
No FDA designations are on record for Nusinersen. Editors add each one with its source.
Filed application
No filed application is on record for Nusinersen. Approvals are listed under FDA actions.
FDA advisory committees
No FDA advisory committee meetings are on record for Nusinersen.
Clinical trials
| Trial | Title | Phase | Status | Enrollment | Primary completion | Results posted |
|---|---|---|---|---|---|---|
| NCT05067790ClinicalTrials.gov record for NCT05067790 (opens in a new tab)ASCEND | A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND) | Phase 3 | Active, not recruiting | 45 (actual) | 24 Sep 2027 (estimated) | No |
| NCT04729907ClinicalTrials.gov record for NCT04729907 (opens in a new tab)ONWARD | A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD) | Phase 3 | Completed | 115 (actual) | 30 Jul 2026 (actual) | No |
| NCT04089566ClinicalTrials.gov record for NCT04089566 (opens in a new tab)DEVOTE | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Phase 3 | Completed | 145 (actual) | 21 Feb 2024 (actual) | Yes First posted Results on ClinicalTrials.gov for NCT04089566 (opens in a new tab) |
Posted results on ClinicalTrials.gov for DEVOTE
Serious adverse events (Part A: From first dose of the study up to Day 389 Part B: From first dose of the study up to Day 399 Part C: From first dose of the study up to Day 361): Part A: 28/28 mg Nusinersen 1 of 6; Part B: Infantile-Onset SMA: 12/12 mg Nusinersen 18 of 25; Part B: Infantile-Onset SMA: 50/28 mg Nusinersen 30 of 50; Part B: Later-Onset SMA: 12/12 mg Nusinersen 4 of 8; Part B: Later-Onset SMA: 50/28 mg Nusinersen 2 of 16; Part C: 50/28 mg Nusinersen 6 of 40 Source: ClinicalTrials.gov results for NCT04089566 (opens in a new tab)· imported Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately. Publications listing DEVOTE (3), newest first
Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies. | ||||||
| NCT02594124ClinicalTrials.gov record for NCT02594124 (opens in a new tab)SHINE | A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies | Phase 3 | Completed | 292 (actual) | 21 Aug 2023 (actual) | Yes First posted Results on ClinicalTrials.gov for NCT02594124 (opens in a new tab) |
Posted results on ClinicalTrials.gov for SHINE Serious adverse events (From Day 1 up to the end of the study (up to 2848 days)): Infantile SMA Onset CS3A 11 of 13; Infantile SMA Onset CS3B Previous Control 23 of 24; Infantile SMA Onset CS3B Previous ISIS 396443 59 of 65; Infantile SMA Onset 232SM202 9 of 12; Later SMA Onset 232SM202 5 of 8; Later SMA Onset CS12 Type 2 12 of 20; Later SMA Onset CS12 Type 3 6 of 25; Later SMA Onset CS4 Previous Control 26 of 42; Later SMA Onset CS4 Previous ISIS 396443 47 of 83 Source: ClinicalTrials.gov results for NCT02594124 (opens in a new tab)· imported Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately. Publications listing SHINE (1), newest first
Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies. | ||||||
| NCT02292537ClinicalTrials.gov record for NCT02292537 (opens in a new tab)CHERISH | A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA) | Phase 3 | Completed | 126 (actual) | 20 Feb 2017 (actual) | Yes First posted Results on ClinicalTrials.gov for NCT02292537 (opens in a new tab) |
Posted results on ClinicalTrials.gov for CHERISH
Serious adverse events (From signing of Informed Consent to the end of the Follow-up period (Month 15)): Sham Procedure 12 of 42; Nusinersen 14 of 84 Source: ClinicalTrials.gov results for NCT02292537 (opens in a new tab)· imported Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately. Publications listing CHERISH (2), newest first
Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies. | ||||||
| NCT02193074ClinicalTrials.gov record for NCT02193074 (opens in a new tab)ENDEAR | A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy | Phase 3 | Terminated | 122 (actual) | 21 Nov 2016 (actual) | Yes First posted Results on ClinicalTrials.gov for NCT02193074 (opens in a new tab) |
Posted results on ClinicalTrials.gov for ENDEAR
Serious adverse events (Screening through Day 394 (± 7 days) or early termination): Control 39 of 41; Nusinersen 61 of 80 Source: ClinicalTrials.gov results for NCT02193074 (opens in a new tab)· imported Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately. Publications listing ENDEAR (5), newest first
Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies. | ||||||
Source: ClinicalTrials.gov, U.S. National Library of Medicine. Posted results are shown as ClinicalTrials.gov prints them; results from company and journal sources are entered by editors and listed separately. Full result tables are on ClinicalTrials.gov.
Trial quality
About CT-VCALoading trial quality…
FDA actions
| Application | Action | Date | Outcome | Review | Source |
|---|---|---|---|---|---|
| NDA209531Action letter for NDA209531 (opens in a new tab) | Supplement 16 Efficacy | Approved | standard | Drugs@FDA via openFDA (opens in a new tab) | |
| NDA209531Action letter for NDA209531 (opens in a new tab) | Supplement 10 Efficacy | Approved | standard | Drugs@FDA via openFDA (opens in a new tab) | |
| NDA209531Action letter for NDA209531 (opens in a new tab) | Supplement 3 Efficacy | Approved | standard | Drugs@FDA via openFDA (opens in a new tab) | |
| NDA209531Action letter for NDA209531 (opens in a new tab) | Original 1 Type 1 - New Molecular Entity | Approved | priority | Drugs@FDA via openFDA (opens in a new tab) |
Data provided by the U.S. Food and Drug Administration.
FDA recalls load here.
FDA drug shortages load here.
Orange Book patents load here.
FAERS adverse event counts load here.
Papers and preprints load here.
Community discussion loads here.
EU status (EMA)
AuthorisedOrphan medicine
Spinraza (EMEA/H/C/004312)
- Authorised
- Therapeutic area
- Muscular Atrophy, Spinal
- Marketing authorisation holder
- Biogen Netherlands B.V.
- Active substance (INN)
- nusinersen
- ATC code
- M09
- Latest EMA decision
Matched by active substance: EMA's INN or active substance equals this drug's name.
EMA page for Spinraza (opens in a new tab)EMA medicine data (opens in a new tab)
Source: European Medicines Agency (EMA), medicines data download. Licence: EMA re-use with acknowledgement. EMA medicines report 2026-10-26.
Related news
No recent headlines mention this drug.
Sources
- Drugs@FDA 209531 (opens in a new tab) Licence: public-domain. Official registry record this listing came from
- ClinicalTrials.gov NCT05067790 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT04729907 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT04089566 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT02594124 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT02292537 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT02193074 (opens in a new tab) Licence: public-domain. Fetched
- Drugs@FDA via openFDA NDA209531 (opens in a new tab) Licence: cc0. Fetched
- 0000875045-25-000047 (opens in a new tab) Licence: public-domain. Entered by an editor, not fetched automatically
Record updated .
Dates come from company and FDA disclosures and can change. Nothing on this page is investment advice.