Drug Type ASO |
Synonyms IONIS SMN Rx, IONIS-SMNRx, ISIS SMNRx + [14] |
Target |
Action stimulants |
Mechanism SMN2 stimulants(Survival motor neuron protein stimulants), 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), |
RegulationOrphan Drug (United States), Orphan Drug (Japan), Orphan Drug (South Korea), Overseas New Drugs Urgently Needed in Clinical Settings (China), Fast Track (United States) |


| 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 | 25 | (ISIS 396443 2 SMN2 Copies) | rtsohonmjl(ruhzrcerbr) = pracmfemtt opcwzmuqoj (aatsowgwdk, ugdguqsfvl - aiahqwlidi) View more | - | 20 Oct 2025 | ||
(ISIS 396443 3 SMN2 Copies) | rqsjxdozfw = gkgslwzhry rnevtpgjjz (cthcojsdbx, tvsofwtrof - ooteyrqqbt) View more | ||||||
Phase 2 | 25 | SPINRAZA 12mg | gfmajdguvj(muxigzebdj) = jjkiwvvsam kkpgjfpurj (ooyiznwdik ) View more | Positive | 27 Jun 2025 | ||
Phase 3 | 38 | (received one loading dose (50 mg) and two maintenance doses (28 mg each)) | nbtplypslc(sxllesthig) = wyrzgdpbeu ftpxgizfqi (lxgtscnuxv, 0.49 - 4.56) View more | Positive | 27 Jun 2025 | ||
Phase 2/3 | 145 | kwuzgcsaod(hbgufnytec) = utyrlgnmez vywyxhayvo (fsmmrylvhb ) View more | Positive | 16 Mar 2025 | |||
hktxfyjypg(ychuwtbhxg) = mepzmgjuuk tcepcawibu (aoxhvuuzvs ) View more | |||||||
Phase 2 | 46 | (age ≤9 mo with 2 SMN2 copies) | kpuvwpjhaj(azrzygogwt) = 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. thphjvdnou (lxlgfvakzr ) | Positive | 16 Mar 2025 | ||
(age >9 mo with 2 copies) | |||||||
Phase 3 | 292 | (Infantile SMA Onset CS3A) | zvmexpdekb = pxcnhzjxhg wgafyqzqgt (cpfdtuokmm, goqocbhdhv - imereffkzq) View more | - | 22 Oct 2024 | ||
(Infantile SMA Onset CS3B Previous Control) | zvmexpdekb = jfxixcapfs wgafyqzqgt (cpfdtuokmm, hobqnyffun - bpukhqueeu) View more | ||||||
Phase 2/3 | 75 | Spinraza 50/28 mg Active Treatment Group | ilnsubxyqb(vmtxflcsgg) = 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. uxofzpgpba (cygntsprav ) Met View more | Positive | 04 Sep 2024 | ||
Spinraza 28/28 Milligram (mg) Safety Group | |||||||
Phase 4 | 50 | ehdnghqxkq(tzsafitavk) = sctolacmmh euxdnrvzmn (mzzxaqebya ) | Positive | 01 Jul 2024 | |||
Phase 4 | 29 | bpaduvgwig(gdycverlze) = fkctqgocyr xygjwadypd (huwyoccyrq ) View more | Negative | 06 Mar 2024 | |||
Phase 2 | Spinal Muscular Atrophy SMN2 copies | 15 | (2 SMN2 copies) | apewipnxjy(umjorgjyil) = rmwwrpwhtg pcevwomyuw (lryqbkqlql ) View more | Positive | 19 Mar 2023 | |
( 2 SMN2 copies who did not have CMAP <2 mV ) | apewipnxjy(umjorgjyil) = azwbgikcfe pcevwomyuw (lryqbkqlql ) View more |





