Risdiplam clinical trials
Risdiplam is named as an intervention in 4 current clinical trials in our USA, UK, Poland, Germany, Belgium, France, Spain, Italy, Netherlands, Israel, Brazil, Taiwan and Australia registers. 1 is recruiting patients now. The most common phase is Phase 4. The sponsor with the most trials is Hoffmann-La Roche (3). The most-studied condition is Muscular Atrophy, Spinal.
Where Risdiplam is approved
All regulators →Of the three regulators tracked here, the first to approve Risdiplam was the US, on 7 Aug 2020. It is approved in 3 of the 3 regulators tracked here.
- 1 EU orphan designation, most recently for treatment of spinal muscular atrophy (26 Feb 2019, withdrawn)
- 2 EU safety letters (DHPC), latest: quality defect for Evrysdi, 12 Jun 2026
From the regulators' own registers: FDA Drugs@FDA, the EMA medicines list and Health Canada's Notice of Compliance database. Dates are the first listed approval of a product containing only this substance; combination products and nationally authorised EU medicines are not counted.
Risdiplam patents and exclusivity
Expiry tracker →Protections still running for marketed products containing Risdiplam, as listed by the regulators. Expired patents and exclusivities, and discontinued products, are not shown.
- 7 listed patents; the last listed expires 15 Apr 2041 (US 12122789).
- Latest patent on the drug substance itself: 15 Apr 2041 (US 12122789).
- Exclusivity ODE-400: Orphan drug exclusivity: treatment of spinal muscular atrophy (sma) in pediatric patients between birth and 2 months of age, until 27 May 2029.
- Exclusivity ODE-334: Orphan drug exclusivity: treatment of spinal muscular atrophy (sma) in patients 2 months of age and older, until 7 Aug 2027.
Reproduced from the FDA Orange Book and Purple Book (checked 8 Nov 2026) and the European Commission's Union Register of medicinal products (CC BY 4.0; the EU expiry date is calculated exactly as the Commission's register displays it). Listed patents and dates are as submitted to or published by the regulator and may not reflect litigation, invalidation, settlements, lapsed fees or later extensions. This is not legal advice or a freedom-to-operate opinion, and it does not say when a generic or biosimilar will launch.
What health systems spend on Risdiplam
All medicines →From NHSBSA Prescription Cost Analysis (Open Government Licence v3.0) and CMS State Drug Utilization Data. Both are gross amounts: neither system publishes its discounts or rebates per drug. Products combining several substances are not counted here.
How Risdiplam works
All drug targets →- SMN2 pre-mRNA positive modulator Positive modulatorTarget: SMN2 survival of motor neuron 2, centromericChEMBL only
- Proximal spinal muscular atrophyPhase 2
- Spinal muscular atrophy, type 1Phase 1/2
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL4297528, CC BY-SA 3.0. Targets checked against the IUPHAR/BPS Guide to PHARMACOLOGY (2026.3.1), ligand 11170, CC BY-SA 4.0. Trial phases are not approvals: approved uses are shown from the regulators' own records where we hold them.
NICE appraisals of Risdiplam
All NICE appraisals →NICE, which decides whether the NHS in England should fund new medicines, has published 2 technology appraisals of Risdiplam. 2 are current, and 2 of these recommend it for at least some patients.
| Appraisal | NICE recommendation | Published | Status |
|---|---|---|---|
| Nusinersen and risdiplam for treating spinal muscular atrophy NICE TA1162 | Recommended Optimised | 4 Jun 2026 | Current |
| Risdiplam for treating spinal muscular atrophy NICE TA755 | Optimised | 16 Dec 2021 Updated 15 Dec 2023 | Current |
NICE decisions apply to the NHS in England. In Ireland the HSE decides on reimbursement, advised by the National Centre for Pharmacoeconomics. Titles, categories and dates link to the guidance on nice.org.uk. © NICE 2026 technology appraisal guidance. Available from www.nice.org.uk/guidance. All rights reserved. Subject to Notice of rights. NICE guidance is prepared for the National Health Service in England. All NICE guidance is subject to regular review and may be updated or withdrawn. NICE accepts no responsibility for the use of its content in this product/publication.
Patient leaflets for Risdiplam
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- IEIreland: 2 products on the HPRA listEach product record links to the HPRA, which publishes the leaflet and SmPC.
- EUEuropean Union: EvrysdiEMA product information: package leaflet and summary of product characteristics in every EU language.
- UKUnited Kingdom: Search MHRA ProductsPatient information leaflets and SmPCs for UK-licensed products.
- USUnited States: Search DailyMedFDA-approved labels, including patient and medication guides.
Leaflets differ between brands and countries; always read the one that comes with your own medicine. This is regulatory information, not medical advice.
Reference identifiers for Risdiplam
- ChEMBL
- CHEMBL4297528
- DrugBank
- DB15305
- PubChem CID
- 118513932
- CAS number
- 1825352-65-5
- FDA UNII
- 76RS4S2ET1
- Wikidata
- Q48969152
- Wikipedia
- Risdiplam
Codes that identify this medicine in other databases, from Wikidata (CC0), matched by its ChEMBL ID. The ATC code is the WHO classification of what the medicine is used for.
Trials by status
Trials by phase
Clinical trials of Risdiplam
4 trialsRegisters list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Trial titles and statuses are copied from the registry as published.
Risdiplam across our data
Ireland · EU · USThe same active substance in our other registers, matched by its exact name. Each entry links to the register record it comes from.
Licensed in Ireland (HPRA)
2 single-substance products containing Risdiplam on the HPRA medicines list.
- Evrysdi 0.75 mg/mL powder for oral solutionRoche Registration GmbH · EU/1/21/1531/001
- Evrysdi 5 mg film-coated tabletsRoche Registration GmbH · EU/1/21/1531/002
Supply shortages
No entry for Risdiplam in the EMA, FDA or German BfArM shortage lists we track.
Conditions studied with Risdiplam
5 conditionsSponsors
lead sponsor per trial| Lead sponsor | Trials |
|---|---|
| Hoffmann-La Roche | 3 |
| Scholar Rock, Inc | 1 |
By country register
A trial with sites in more than one country appears in each of those registers.
Related compounds
Questions about Risdiplam trials
How many clinical trials are testing Risdiplam?
4 current trials in our USA, UK, Poland, Germany, Belgium, France, Spain, Italy, Netherlands, Israel, Brazil, Taiwan and Australia registers name Risdiplam (also registered as RO7034067) as a drug or biological intervention, as of the registry sync on 9 November 2026. Completed and withdrawn studies are not counted.
Are Risdiplam trials recruiting patients?
Yes. 1 of the 4 trials is recruiting: 1 with sites in USA, 1 with sites in UK, 1 with sites in Belgium, 1 with sites in France, 1 with sites in Spain, 1 with sites in Italy and 1 with sites in Netherlands. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.
Who sponsors Risdiplam clinical trials?
Hoffmann-La Roche (3) and Scholar Rock, Inc (1). The number in brackets is how many trials each organisation leads.
Is Risdiplam licensed in Ireland?
Yes. The HPRA medicines list includes 2 single-substance products containing Risdiplam, for example Evrysdi 0.75 mg/mL powder for oral solution (Roche Registration GmbH). A licence does not mean a product is currently marketed or reimbursed; see each product's record.
Is there a Risdiplam shortage?
Not in the lists we track: Risdiplam has no entry in the EMA, FDA or German BfArM shortage registers as of their latest sync.
Where does this information come from?
Every trial, status, phase and sponsor on this page is copied from public ClinicalTrials.gov records by a daily sync. Nothing here describes how Risdiplam works or whether it is effective, and it is not medical advice.
How this page is built
Everything on this page is copied from public ClinicalTrials.gov registry records — it is not written from memory, and nothing here describes how Risdiplam works or whether it is effective. Each trial links to its own record so you can check it yourself.
- ClinicalTrials.gov (U.S. National Library of Medicine) ↗ Trial IDs, titles, intervention names and synonyms, phase, overall status, lead sponsor and conditions for every trial on this page
This page is built automatically: a daily sync job reads the registry records behind our country trial registers, collects every trial that lists Risdiplam (or a registered synonym) as a drug or biological intervention, and tabulates the registry fields directly. Placebos and generic comparators are excluded. There is no AI drafting and no per-compound editorial pass, and the page has not been clinically reviewed. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The ClinicalTrials.gov data on this page was last retrieved on 9 Nov 2026. How every register is built: methodology · fixes we have made: corrections log.