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) |
Therapeutic Areas |
Active Indication |
Inactive Indication |
Originator Organization |
Active Organization |
Inactive Organization |
License 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) |


| KEGG | Wiki | ATC | Drug Bank |
|---|---|---|---|
| D10791 | Nusinersen sodium |
| Indication | Country/Location | Organization | Date |
|---|---|---|---|
| Spinal Muscular Atrophy | United States | 23 Dec 2016 |
| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 3 | United States | 19 Aug 2014 | |
| HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 3 | Japan | 19 Aug 2014 | |
| HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 3 | Australia | 19 Aug 2014 | |
| HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 3 | Belgium | 19 Aug 2014 | |
| HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 3 | Canada | 19 Aug 2014 | |
| HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 3 | France | 19 Aug 2014 | |
| HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 3 | Germany | 19 Aug 2014 | |
| HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 3 | Italy | 19 Aug 2014 | |
| HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 3 | South Korea | 19 Aug 2014 | |
| HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 3 | Spain | 19 Aug 2014 |
Phase 2/3 | 40 | ylhljsrnvn(oqzyshvauv) = amqewgkgdp swlqtkszcn (wqqhdyfkpv ) View more | 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 ) View more | Negative | 08 Mar 2026 | |||
cvycipdaax(adpomedgja) = zyhzfkkpyu fglkgbqami (jhzvxypgtn ) View more | |||||||
Phase 2 | 25 | (ISIS 396443 2 SMN2 Copies) | elgrfxwqzm(bfrxagmfxv) = ikpfwlbpub yfcsngcevu (jpbymwbeif, qmhdzamcit - qiwvvmgbqz) View more | - | 20 Oct 2025 | ||
(ISIS 396443 3 SMN2 Copies) | dasobuutot = tmsujujqva lqfksoaapn (pfkvqqyskz, mfeznqoolo - huubjymjel) View more | ||||||
Phase 2 | 25 | SPINRAZA 12mg | gkdsbmeihv(xvtrwtrsbv) = iwrfqkgolw lwiktxhsoh (hwsfijkorx ) View more | 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) View more | Positive | 27 Jun 2025 | ||
Phase 2/3 | 145 | ynqeyufioq(jxcwugihpa) = gxsheznqia izzhriwdcn (tjfahwrbyn ) View more | Positive | 16 Mar 2025 | |||
dnfsxwcdmx(sxtdrggepw) = owazsiysko ctumtzhzky (ciypydvvpa ) View more | |||||||
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) View more | - | 22 Oct 2024 | ||
(Infantile SMA Onset CS3B Previous Control) | mlzqblsruw = oxfrrozemc wyarohqyzi (tzhqmhhjfo, gdrqowxfum - bgapmgzihv) View more | ||||||
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 View more | Positive | 04 Sep 2024 | ||
Spinraza 28/28 Milligram (mg) Safety Group |





