risdiplam
Trade name: Evrysdi
Small moleculeapprovedOrphan Drug FDAFast Track FDA
Approved
Aug 7, 2020
Risdiplam, sold under the brand name Evrysdi, is a medication used to treat spinal muscular atrophy (SMA) and is the first oral medication approved to treat this disease by the US Food and Drug Administration (FDA). — Wikipedia
Clinical trial activity
18 trials · 4 clinical orgs · 2 marketing orgs
Phase 1
6
Phase 2
6
Phase 3
1
Phase 4
4
Earliest trial started Jan 1, 2016 (NCT02633709)
Timeline
2010s
Indications
Mechanism of action
- Survival motor neuron proteinACTIVATOR
Survival motor neuron protein activator
- SMN2 pre-mRNAPOSITIVE MODULATOR
SMN2 pre-mRNA positive modulator
May cause alternative splicing of additional non-target genes inc. FOXM1 and MADD which may contribute to adverse effects.
Approval history
- approvedPriority reviewFast trackAug 7, 2020
Chemistry & pharmacology
Loading structure…
SMILES
Cc1cn2nc(-c3cc(=O)n4cc(N5CCNC6(CC6)C5)ccc4n3)cc(C)c2n1- Mol. weight
- 401.47 g/mol
- Lipinski Ro5
- Pass
- Rule of 3
- No
- Chirality
- Achiral Molecule
- Inorganic
- No
- Polymer
- No
- Delivery
- Oral
- Availability
- Prescription Only
Oral
Yes
Parenteral
No
Topical
No
Sources
- WikipediaRisdiplam ↗
- ChEMBLCHEMBL4297528 ↗
Also known as
- ro7034067
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT07047144 | Phase 2 | Sep 15, 2025 | Scholar Rock, Inc. |
| NCT05861986 | Phase 4 | Feb 7, 2024 | Roche |
| NCT05808764 | Phase 2 | Jan 15, 2024 | Roche |
| NCT05861999 | Phase 4 | Dec 29, 2023 | Roche |
| NCT05522361 | Phase 4 | Sep 15, 2022 | Clinic for Special Children, Genentech |
| NCT05232929 | Phase 4 | Mar 29, 2022 | Genentech, Roche |
| NCT05115110 | Phase 2/Phase 3 (Phase 3) | Feb 14, 2022 | Roche |
| NCT04718181 | Phase 1 | Feb 1, 2021 | Roche |
| NCT03988907 | Phase 1 | Jun 21, 2019 | Roche |
| NCT03779334 | Phase 2 | Jun 14, 2019 | Roche |
| NCT03920865 | Phase 1 | May 16, 2019 | Roche |
| NCT03040635 | Phase 1 | Mar 22, 2017 | Roche |
| NCT03032172 | Phase 2 | Mar 2, 2017 | Roche |
| NCT03036501 | Phase 1 | Jan 3, 2017 | Roche |
| NCT02913482 | Phase 2 | Dec 24, 2016 | Roche |
| NCT02908685 | Phase 2 | Oct 20, 2016 | Roche |
| NCT02633709 | Phase 1 | Jan 1, 2016 | Roche |
| NCT04256265 | N/A | — | Genentech, Roche |
Showing 18 of 18 trials
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Organizations
Research & Development (4)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Roche | For profit | 16 | 14 | 5 | 2016 |
| Genentech | For profit | 3 | 2 | 2 | 2022 |
| Clinic for Special Children | Academic/Hospital | 1 | 1 | 1 | 2022 |
| Scholar Rock, Inc. | For profit | 1 | 1 | 1 | 2025 |
4 organizations
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