Evrysdi
An oral liquid medication used daily to treat spinal muscular atrophy.
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- 1 EU orphan designation, most recently for treatment of spinal muscular atrophy (26 Feb 2019, withdrawn)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 12 Oct 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), 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.
What Evrysdi is used for
Risdiplam (Evrysdi) is an oral liquid medication used daily to treat spinal muscular atrophy. It belongs to the SMN2 splicing modifier class.
Related clinical trials recruiting now
Compare Evrysdi with
Explore more
How this page is built
The facts on this page are pulled directly from official U.S. FDA datasets — they are not written from memory. Each field below names the dataset it came from, so you can check it yourself.
- FDA Drug Label (openFDA) ↗ Indications, mechanism of action and pharmacologic class
- Drugs@FDA ↗ First FDA approval date and the company holding the application
- FDA Drug Shortage Database ↗ Current US supply / shortage status
Plain-English summaries and drug-class explainers are written and reviewed by Sreepriya Prasannan (MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany). Data is retrieved automatically from the sources above and cross-checked with AI-assisted verification (Anthropic's Claude) — brand and generic names are matched against the exact FDA product record so that a combination product or a different formulation cannot be mistaken for the drug on this page. An editor reviews the result before publication. We describe this in full in our editorial standards and corrections policy. The FDA data on this page was last retrieved on 6 Oct 2026. How every register is built: methodology · fixes we have made: corrections log.