Drugs / Nusinersen
Nusinersen
also known as
BIIB-058 · ISIS 396443 · ISIS-396443 SODIUM · ISIS-SMNRx · NUSINERSEN SODIUM · Spinraza
Antisense oligonucleotide targets SMN2
Regulatory milestones approvals, filings & regulatory actions · 5 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Label expansions (3) | US (FDA) | SPINRAZA | — | 2018-05-14 – 2026-03-27 | fda.gov ↗ |
| Approved | EU (EMA) | Spinraza | — | 2017-05-30 | europa.eu ↗ |
| Approved | US (FDA) | SPINRAZA | — | 2016-12-23 | fda.gov ↗ |
Trials 18 · a red edge is where a trial was stopped
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 01 trial | ||||||
| Phase 0 | NCT04050852 | Jul 2019 → Apr 2021 | spinal muscular atrophy | NYU Langone Health | Withdrawn | No outcome recorded |
| Phase 14 trials | ||||||
| Phase 1 | NCT06555419 | Jan 2025 → Jun 2027 expected | spinal muscular atrophy | Biogen | Recruiting | No outcome recorded |
| Phase 1 | NCT02052791 | Jan 2014 → Jan 2017 | spinal muscular atrophy | Biogen | Completed | No outcome recorded |
| Phase 1 | NCT01780246 | Jan 2013 → Feb 2014 | spinal muscular atrophy | Biogen | Completed | No outcome recorded |
| Phase 1 | NCT01494701 | Nov 2011 → Jan 2013 | spinal muscular atrophy | Biogen | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT01703988 | Oct 2012 → Jan 2015 | spinal muscular atrophy | Biogen | Completed | No outcome recorded |
| Phase 23 trials | ||||||
| Phase 2 | NCT02462759 | Aug 2015 → Sep 2018 | spinal muscular atrophy | Biogen | Terminated | No outcome recorded |
| Phase 2 | NCT02386553 | May 2015 → Dec 2024 | spinal muscular atrophy | Biogen | Completed | No outcome recorded |
| Phase 2 | NCT01839656 | May 2013 → Aug 2017 | spinal muscular atrophy | Biogen | Completed | No outcome recorded |
| Phase 36 trials | ||||||
| Phase 3 | NCT05067790 | Jan 2022 → Sep 2027 expected | spinal muscular atrophy | Biogen | Active not recruiting | No outcome recorded |
| Phase 3 | NCT04729907 | Apr 2021 → Jul 2026 overdue | spinal muscular atrophy | Biogen | Active not recruiting | No outcome recorded |
| Phase 3 | NCT04089566 | Mar 2020 → Feb 2024 | spinal muscular atrophy | Biogen | Completed | No outcome recorded |
| Phase 3 | NCT02594124 | Nov 2015 → Aug 2023 | spinal muscular atrophy | Biogen | Completed | No outcome recorded |
| Phase 3 | NCT02292537 | Nov 2014 → Feb 2017 | spinal muscular atrophy | Biogen | Completed | No outcome recorded |
| Phase 3 | NCT02193074 | Aug 2014 → Nov 2016 | spinal muscular atrophy | Biogen | Terminated | No outcome recorded |
| Phase 41 trial | ||||||
| Phase 4 | NCT04488133 | Jan 2021 → Oct 2025 | spinal muscular atrophy | Biogen | Completed | No outcome recorded |
| Phase not applicable1 trial | ||||||
| — | NCT04576494 | Jan 2022 → Jan 2023 | spinal muscular atrophy | Reims University Hospital | Completed | No outcome recorded |
| Phase not stated1 trial | ||||||
| — | NCT02865109 | — | spinal muscular atrophy, type 1 | Biogen | No longer available | No outcome recorded |
Also used as a comparator or background therapy in 1 trial
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 2 | NCT07047144 Background | Sep 2025 → Nov 2028 expected | spinal muscular atrophy, type II, spinal muscular atrophy, type III | Scholar Rock, Inc. | Recruiting | No outcome recorded |
Evidence & citations 11 cited values
Every value below carries the sentence it was read from. 22 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Nusinersen | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05067790 ↗ |
| Known as | BIIB-058 | ChEMBL registry synonym — accepted as the source's own label CHEMBL3301572 ↗ |
| Known as | ISIS 396443 | ChEMBL registry synonym — accepted as the source's own label CHEMBL3301572 ↗ |
| Known as | ISIS-396443 SODIUM | ChEMBL registry synonym — accepted as the source's own label CHEMBL3301572 ↗ |
| Known as | ISIS-SMNRx | “Nusinersen (ISIS-SMNRx or ISIS 396443) is an antisense oligonucleotide drug administered intrathecally to treat spinal muscular atrophy.” PMID 26823478 ↗ Jan 2016 |
| Known as | NUSINERSEN SODIUM | ChEMBL registry synonym — accepted as the source's own label CHEMBL3301572 ↗ |
| Known as | Spinraza | ChEMBL registry synonym — accepted as the source's own label CHEMBL3301572 ↗ |
| Action | Restore | “thus increase the amount of functional survival motor neuron (SMN) protein that is deficient in patients with spinal muscular atrophy” PMID 27939059 ↗ Dec 2016 |
| Modality | Antisense oligonucleotide | “is an antisense oligonucleotide drug” PMID 26823478 ↗ Jan 2016 |
| Route | Intrathecal | “administered intrathecally” NCT01494701 ↗ |
| Target | SMN2 | “specifically designed to alter splicing of SMN2 pre-mRNA” PMID 27939059 ↗ Dec 2016 |