Product Description
RisdiplamÊ(RG7916,ÊRO7034067) is a highly potent, selective and orally activeÊsmall moleculeÊexperimental drugÊbeing developed byÊF. Hoffmann-La Roche,ÊPTC TherapeuticsÊandÊSMA FoundationÊto treatÊspinal muscular atrophyÊ(SMA). It is aÊpyridazineÊderivative that works by increasing the amount of functionalÊsurvival of motor neuronÊprotein produced by theÊSMN2ÊgeneÊthroughÊmodifying its splicing pattern (Sourced from: https://newdrugapprovals.org/tag/ro-7034067/)
Mechanisms of Action: SMN2 Modulator
Novel Mechanism: Yes
Modality: Small Molecule
Route of Administration: Oral,Injection,Nasal
FDA Designation: None *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Hoffmann-La Roche
Company Location: BASEL SWITZERLAND V8
Company CEO: Severin Schwan
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Australia, Belgium, Brazil, Canada, Croatia, France, Germany, Italy, Japan, Netherlands, Poland, Portugal, Russia, Saudi Arabia, Serbia, Spain, Switzerland, Turkey, United Kingdom, United States, Unknown Location
Active Clinical Trial Count:
Highest Development Phases
Phase 3: Muscular Atrophy, Spinal
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| BN42644 | P3 |
Unknown Status |
Muscular Atrophy, Spinal |
2029-03-27 |
|
| 2021-003417-19 | P3 |
Active, not recruiting |
Muscular Atrophy, Spinal |
2026-08-04 |
|
| MANATEE | P3 |
Active, not recruiting |
Muscular Atrophy, Spinal |
2026-06-27 |
|
| Jewelfish | P2 |
Active, not recruiting |
Muscular Atrophy, Spinal |
2024-12-27 |