Drug Type Antisense oligonucleotides |
Synonyms IONIS-SMNRx, ISIS-SMNRx, nusinersen + [10] |
Target |
Mechanism SMN2 stimulants(Survival motor neuron protein stimulants), RNA interference |
Therapeutic Areas |
Active Indication |
Inactive Indication |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhaseApproved |
First Approval Date US (23 Dec 2016), |
RegulationFast Track (US), Orphan Drug (US), Orphan Drug (JP), Overseas New Drugs Urgently Needed in Clinical Settings (CN) |
KEGG | Wiki | ATC | Drug Bank |
---|---|---|---|
D10791 | Nusinersen sodium |
Indication | Country/Location | Organization | Date |
---|---|---|---|
Neuromuscular Diseases | CN | 22 Feb 2019 | |
Spinal Muscular Atrophy | US | 23 Dec 2016 |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 3 | US | 19 Aug 2014 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 3 | JP | 19 Aug 2014 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 3 | AU | 19 Aug 2014 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 3 | BE | 19 Aug 2014 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 3 | CA | 19 Aug 2014 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 3 | FR | 19 Aug 2014 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 3 | DE | 19 Aug 2014 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 3 | IT | 19 Aug 2014 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 3 | KR | 19 Aug 2014 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 3 | ES | 19 Aug 2014 |
RESPOND (GlobeNewswire) Manual | Phase 4 | 29 | pcbcjetfcd(hulxwiscte) = ifeyqrwdfw bruxaogpwe (gvgobiipjm ) View more | Negative | 06 Mar 2024 | ||
Not Applicable | - | goytkqdiya(zjgmhalnic) = bjecfekntm qefqzxrvup (xqeseaodfo ) View more | Positive | 03 Mar 2024 | |||
goytkqdiya(zjgmhalnic) = gzitfaaobn qefqzxrvup (xqeseaodfo ) View more | |||||||
Not Applicable | - | cbhtmcqlaz(ixafucuzaj) = qsavhptiyh svaxauybji (ufazxcviax ) | - | 03 Mar 2024 | |||
Not Applicable | 49 | nudzawavaq(qqtwhrfamq) = acovgxjdcw zamhfeicre (oyeeuhxmbc ) | Positive | 03 Mar 2024 | |||
Phase 2 | Spinal Muscular Atrophy SMN2 copies | 15 | (2 SMN2 copies) | ohpfhepukm(reqigzcolg) = yuvtngrlyd lemeamoupi (xuzbooxlpt ) View more | Positive | 19 Mar 2023 | |
( 2 SMN2 copies who did not have CMAP <2 mV ) | ohpfhepukm(reqigzcolg) = xzsxvcocoe lemeamoupi (xuzbooxlpt ) View more | ||||||
Phase 4 | 34 | vofkqoyqhh(kvawonbynu) = Upper respiratory tract infection (n=6) and viral upper respiratory tract infection (n=5) oyvhipbzpb (atlgnshqox ) | Negative | 19 Mar 2023 | |||
Pubmed Manual | Not Applicable | 401 | (infantile-onset SMA) | eoauojfklo(hjhaedfizz) = rdgwjwmgve xkotgwcfbt (fhqmqlrdhr ) | Positive | 05 Jul 2022 | |
(later-onset SMA) | eoauojfklo(hjhaedfizz) = ozfhnpbdyb xkotgwcfbt (fhqmqlrdhr ) | ||||||
Not Applicable | - | qcawiykzuo(wwxhtflctd) = cqkjgxybmi bhbxktlzai (nqfhhetczw ) View more | Positive | 03 May 2022 | |||
Not Applicable | 190 | fwowzmxtpz(mttrxylvxo) = 3% zdwlfdypth (onkjtmyifm ) View more | Positive | 13 Mar 2022 | |||
Phase 4 | Spinal Muscular Atrophy SMN2 copies | 9 | duatsvskrn(ugctvvmwdd) = 4 participants, 7 events [ear infection, viral gastroenteritis, parainfluenza virus infection, pneumonia, upper respiratory tract infection (URTI), viral URTI] rknlwxnrix (nztsxsbngb ) View more | Positive | 13 Mar 2022 |