nusinersen
Trade name: Spinraza
Approved
Dec 23, 2016
Nusinersen, marketed as Spinraza, is a medication used in treating spinal muscular atrophy (SMA), a rare neuromuscular disorder. In December 2016, it became the first approved drug used in treating this disorder. — Wikipedia
Clinical trial activity
20 trials · 6 clinical orgs · 1 marketing orgs
Earliest trial started Nov 30, 2011 (NCT01494701)
Timeline
2010s
- Jan 1, 2011
Earliest Phase 1 Sponsor(trial)
- Apr 18, 2011
Orphan Drug Designation
- Jan 1, 2013
Earliest Phase 2 Sponsor(trial)
- Jan 1, 2014
Earliest Phase 3 Sponsor(trial)
- Dec 23, 2016
Biogen-IDEC — Earliest FDA Approval
- Dec 23, 2016
Biogen-IDEC — NDA Secondary Org
- Dec 23, 2016
Biogen-IDEC — NDA Organization
Indications
Approved for
Mechanism of action
- SMN2 pre-mRNAPOSITIVE MODULATOR
SMN2 pre-mRNA positive modulator
SPINRAZA was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein
- SMN2 pre-mRNAMODULATOR
SMN2 pre-mRNA modulator
SPINRAZA was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein
Approval history
- approvedPriority reviewFast trackDec 23, 2016
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Availability
- Prescription Only
Oral
No
Parenteral
No
Topical
No
Sources
- WikipediaNusinersen ↗
- NCATS5Z9SP3X666 ↗
- ChEMBLCHEMBL3833342 ↗
- ChEMBLCHEMBL3301572 ↗
Also known as
- isis 396443
- isis 396443
- isis-396443
- isis-396443
- isis-smnrx
- isis-smnrx
- nusinersen
- nusinersen
- nusinersen
- nusinersen
- nusinersen
- nusinersen sodium
- nusinersen sodium
- nusinersen sodium
- spinraza
- spinraza
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT07047144 | Phase 2 | Sep 15, 2025 | Scholar Rock, Inc. |
| NCT06555419 | Phase 1 | Dec 16, 2024 | Biogen |
| NCT05067790 | Phase 3 | Nov 22, 2021 | Biogen |
| NCT04729907 | Phase 3 | Apr 19, 2021 | Biogen |
| NCT04488133 | Phase 4 | Feb 26, 2021 | Biogen |
| NCT04576494 | N/A | Dec 31, 2020 | University of Reims |
| NCT04089566 | Phase 2/Phase 3 (Phase 3) | Feb 26, 2020 | Biogen |
| NCT04159987 | N/A | Nov 30, 2019 | University of Nice Sophia Antipolis |
| NCT04050852 | Early Phase 1 (Phase 1) | Jul 17, 2019 | New York University, Winthrop University |
| NCT02865109 | N/A | Aug 10, 2016 | Biogen |
| NCT02594124 | Phase 3 | Nov 4, 2015 | Biogen |
| NCT02462759 | Phase 2 | Aug 19, 2015 | Biogen |
| NCT02386553 | Phase 2 | May 20, 2015 | Biogen |
| NCT02292537 | Phase 3 | Nov 1, 2014 | Biogen |
| NCT02193074 | Phase 3 | Jul 1, 2014 | Biogen |
| NCT02052791 | Phase 1 | Jan 1, 2014 | Biogen |
| NCT01839656 | Phase 2 | May 1, 2013 | Biogen |
| NCT01780246 | Phase 1 | Jan 1, 2013 | Biogen |
| NCT01703988 | Phase 1/Phase 2 (Phase 2) | Oct 1, 2012 | Biogen |
| NCT01494701 | Phase 1 | Nov 30, 2011 | Biogen |
Organizations
Research & Development (6)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Biogen | For profit | 16 | 16 | 5 | 2011 |
| New York University | Academic/Hospital | 1 | 1 | 1 | 2019 |
| Scholar Rock, Inc. | For profit | 1 | 1 | 1 | 2025 |
| University of Nice Sophia Antipolis | Academic/Hospital | 1 | 1 | 1 | 2019 |
| University of Reims | Academic/Hospital | 1 | 1 | 1 | 2020 |
| Winthrop University | Academic/Hospital | 1 | 0 | 1 | 2019 |
Marketing (2)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| Biogen-IDEC | For profit | NDA2 | Dec 23, 2016 |
| Biogen-IDEC | For profit | NDA | Dec 23, 2016 |