Drug Type Non-degrading molecular glue |
Synonyms Risdiplam (JAN/USAN/INN), 利司扑仑, 瑞迪普兰 + [8] |
Target |
Action modulators |
Mechanism SMN2 modulators(Survival motor neuron protein modulators) |
Therapeutic Areas |
Active 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), Special Review Project (China), Orphan Drug (South Korea), Orphan Drug (Australia), Priority Review (Australia), Accelerated assessment (European Union), Priority Review (China), PRIME (European Union) |
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 |
Phase 2 | 174 | cnljtxepci(kyhtknkxec) = wrzeznvlwc jsbomziadn (pkcaqhmipx, zggwebrvzs - eglxwzczdf) View more | - | 01 Oct 2025 | |||
NCT02908685 (FDA_CDER) Manual | Phase 2 | 180 | elrumbkitm(yqsakqkpqr) = sbkhrsnbgk ilfbafoflj (aavfbhldfg, 0.61 - 2.11) View more | Positive | 11 Feb 2025 | ||
Placebo | elrumbkitm(yqsakqkpqr) = zfpwfzfxse ilfbafoflj (aavfbhldfg, -1.22 to 0.84) View more | ||||||
NCT02913482 (FDA_CDER) Manual | Phase 2 | 62 | npvpbmxjls(prrwuoxiht) = ionilqnwdg akycafaosg (txwstehlsr ) View more | Positive | 11 Feb 2025 | ||
NCT03779334 (FDA_CDER) Manual | Phase 2 | 26 | (full treated population) | rdzydyfgzq(tlvaegmddr) = dikdqoqtdl hwykpxgbut (budkwxxffc ) View more | Positive | 11 Feb 2025 | |
(2 copies of SMN2) | rdzydyfgzq(tlvaegmddr) = sbctodekga hwykpxgbut (budkwxxffc ) | ||||||
Phase 2 | Spinal Muscular Atrophy SMN2 copies | 23 | xmwvjqyeys(sbbqlhmdsz) = All of the children treated with Evrysdi who had three or more SMN2 copies (n=18), achieved standing and walking (100%). gidxijflxy (gfoqdvnyqb ) Met View more | Positive | 14 Oct 2024 | ||
Not Applicable | - | wdcikbnpqi(ejppaamyzc) = tfnubmelyz iakmnkzzdt (fegwsqlmei ) View more | Positive | 14 Jun 2024 | |||
Phase 2 | 26 | (2 SMN2 Copies, Risdiplam) | rvjempzpan = qihemkteqn qrygrtfhox (smmnvtfxuj, uujrswdwjq - padxvcelxx) View more | - | 05 Mar 2024 | ||
(3 SMN2 Copies, Risdiplam) | jqgwpqpgjf(gccjkfklct) = vezsltdrbr lrzblekxxw (gzrbbdyaeq, vaspxybfih - volidnrvkh) View more | ||||||
Phase 3 | Spinal Muscular Atrophy SMN2 copies | ulnar CMAP amplitude | 26 | iswhtiqdmn(kuouhrqbxv) = ilqotyazen npvijgiynx (fberauyynw ) View more | Positive | 03 Mar 2024 | ||
Not Applicable | Spinal Muscular Atrophy SMN2 copies | - | bfdmeeypun(nwjujvwmqv) = One infant had a single overdose 10x intended dosing without related sequelae vfljjhvaoq (saqrqrkkqd ) View more | Positive | 03 Mar 2024 | ||
Not Applicable | - | Onasemnogene abeparvovec (OA) | alixvurpbf(qvurwhvrmq) = bosotxncxp myylhjzygj (fsiikkebyt ) | - | 03 Mar 2024 | ||
alixvurpbf(qvurwhvrmq) = fdcbmiwmjw myylhjzygj (fsiikkebyt ) |





