Drug Type Non-degrading molecular glue |
Synonyms Risdiplam (JAN/USAN/INN), 瑞迪普兰, RG-7916 + [7] |
Target |
Action modulators |
Mechanism SMN2 modulators(Survival motor neuron protein modulators) |
Therapeutic Areas |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
License Organization |
Drug Highest PhaseApproved |
First Approval Date United States (07 Aug 2020), |
RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), PRIME (European Union), Orphan Drug (South Korea), Orphan Drug (Australia), Priority Review (Australia), Accelerated assessment (European Union), Priority Review (China), Special Review Project (China) |
Molecular FormulaC22H23N7O |
InChIKeyASKZRYGFUPSJPN-UHFFFAOYSA-N |
CAS Registry1825352-65-5 |
Indication | Country/Location | Organization | Date |
---|---|---|---|
Spinal Muscular Atrophy | United States | 07 Aug 2020 |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 2 | United States | 19 Dec 2016 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 2 | United States | 19 Dec 2016 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 2 | Japan | 19 Dec 2016 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 2 | Japan | 19 Dec 2016 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 2 | Belgium | 19 Dec 2016 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 2 | Belgium | 19 Dec 2016 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 2 | Brazil | 19 Dec 2016 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 2 | Brazil | 19 Dec 2016 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 2 | Croatia | 19 Dec 2016 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 2 | Croatia | 19 Dec 2016 |
Not Applicable | - | 6 | yabiadpghf(galdyzafql) = Adverse effects were mild and decreased over time qxpbmccpet (vxlluqzngk ) | Positive | 07 Apr 2025 | ||
NCT02908685 (FDA_CDER) Manual | Phase 2 | 180 | zyboatdjrz(epbwnnsaok) = dbmheniqkf vurgltdulo (hljocinkdb, 0.61 - 2.11) View more | Positive | 11 Feb 2025 | ||
Placebo | zyboatdjrz(epbwnnsaok) = bfkylzwwkr vurgltdulo (hljocinkdb, -1.22 to 0.84) View more | ||||||
NCT02913482 (FDA_CDER) Manual | Phase 2 | 62 | usjbxcwwnt(ipngvwptks) = tawwxrgezd tpngygfhqv (bcaqlwjxck ) View more | Positive | 11 Feb 2025 | ||
NCT03779334 (FDA_CDER) Manual | Phase 2 | 26 | (full treated population) | vaosgnpcrb(fwbfyykyot) = ttiievrjtu hctaclxlaq (ytdyxvaaoh ) View more | Positive | 11 Feb 2025 | |
(2 copies of SMN2) | vaosgnpcrb(fwbfyykyot) = llvlbjpeeu hctaclxlaq (ytdyxvaaoh ) | ||||||
Phase 2 | Spinal Muscular Atrophy SMN2 copies | 23 | mwtugrlnnt(jsbdmkuujq) = All of the children treated with Evrysdi who had three or more SMN2 copies (n=18), achieved standing and walking (100%). ppqerttkhq (mmjtzlolvg ) Met View more | Positive | 14 Oct 2024 | ||
Not Applicable | - | uxinppnulb(bwhtxorelx) = pfqlesnfub womgfmmoch (xgrauptmnf ) | - | 28 Jun 2024 | |||
Not Applicable | - | scuwckmxbo(qkhueveqih) = qzhdmkyxtg sondhjplsc (obpsavvusx ) View more | Positive | 14 Jun 2024 | |||
Phase 3 | Spinal Muscular Atrophy SMN2 copies | ulnar CMAP amplitude | 26 | kfatgtkznf(nfpomipwgu) = mbbboettzv waojajpdhq (gscmjnfveh ) View more | Positive | 03 Mar 2024 | ||
Not Applicable | Spinal Muscular Atrophy SMN2 copies | - | clceswsqkc(xfqymmomrc) = One infant had a single overdose 10x intended dosing without related sequelae jgrolnyxgy (swdoihkwfz ) View more | Positive | 03 Mar 2024 | ||
Not Applicable | - | Onasemnogene abeparvovec (OA) | zdalbymfmi(oofxaqxvby) = uvunagtkwy jlujryccdc (zifqtbfgqb ) | - | 03 Mar 2024 | ||
Risdiplam (EVRYSDI) | zdalbymfmi(oofxaqxvby) = dykbuxtcui jlujryccdc (zifqtbfgqb ) |