Overview
Risdiplam is an orally bioavailable mRNA splicing modifier used for the treatment of spinal muscular atrophy (SMA). It increases systemic SMN protein concentrations by improving the efficiency of SMN2 gene transcription. This mechanism of action is similar to its predecessor nusinersen, the biggest difference being their route of administration: nusinersen requires intrathecal administration, as does the one-time gene therapy onasemnogene abeparvovec, whereas risdiplam offers the ease of oral bioavailability. Risdiplam was approved by the FDA in August 2020 for the treatment of spinal muscular atrophy (SMA). Set to be substantially cheaper than other available SMA therapies, risdiplam appears to provide a novel and relatively accessible treatment option for patients with SMA regardless of severity or type.
Indication
Risdiplam is indicated for the treatment of spinal muscular atrophy (SMA).
Associated Conditions
- Spinal Muscular Atrophy (SMA)
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
|---|---|---|---|---|---|
2025/07/02 | Not Applicable | Not yet recruiting | |||
2025/05/18 | N/A | Recruiting | Newcastle-upon-Tyne Hospitals NHS Trust | ||
2023/05/17 | Phase 4 | Recruiting | |||
2023/05/17 | Phase 4 | Recruiting | |||
2023/04/11 | Phase 2 | Recruiting | |||
2022/08/31 | Phase 4 | Active, not recruiting | Clinic for Special Children | ||
2022/02/10 | Phase 4 | Active, not recruiting | |||
2022/02/02 | N/A | Recruiting | Bakri Elsheikh | ||
2021/11/10 | Phase 2 | Active, not recruiting | |||
2021/01/22 | Phase 1 | Completed |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
|---|---|---|---|---|---|
| Genentech Inc. | 50242-175 | ORAL | 0.75 mg in 1 mL | 2/29/2024 |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
|---|---|---|---|
Authorised | 3/26/2021 | ||
Authorised | 3/26/2021 | ||
Authorised | 3/26/2021 |
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| EVRYSDI POWDER FOR ORAL SOLUTION 0.75 MG/ML | SIN16349P | POWDER, FOR SOLUTION | 60 mg | 10/20/2021 |
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
|---|---|---|---|---|---|
| No NMPA approvals found for this drug. | |||||
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
|---|---|---|---|---|---|
| EVRYSDI POWDER FOR ORAL SOLUTION 0.75MG/ML | N/A | N/A | N/A | 10/7/2021 |
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
|---|---|---|---|---|---|
| EVRYSDI risdiplam 0.75 mg/mL powder for oral solution bottle | 340350 | Medicine | A | 6/2/2021 |
Health Canada Drug Approvals
Approved Product | Company | DIN | Dosage Form | Strength | Market Date |
|---|---|---|---|---|---|
| EVRYSDI | Hoffmann-La Roche Limited | 02514931 | Powder For Solution - Oral | 0.75 MG / ML | 5/19/2021 |
CIMA AEMPS Drug Approvals
Approved Product | Company | Registration Number | Pharmaceutical Form | Prescription Type | Status |
|---|---|---|---|---|---|
| No CIMA AEMPS (Spain) approvals found for this drug. | |||||
Philippines FDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Philippines FDA approvals found for this drug. | |||||
Saudi SFDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Saudi SFDA approvals found for this drug. | |||||
Malaysia NPRA Drug Approvals
Approved Product | Company | Registration Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Malaysia NPRA approvals found for this drug. | |||||
UK EMC Drug Information
Medicine Name | MA Holder | MA Number | Pharmaceutical Form | Active Ingredient | Authorization Date |
|---|---|---|---|---|---|
| No UK EMC drug information found for this drug. | |||||
Help Us Improve
Your feedback helps us provide better drug information and insights.