Spinraza
An intrathecal therapy for spinal muscular atrophy.
Where Nusinersen is approved
All regulators →Of the three regulators tracked here, the first to approve Nusinersen was the US, on 23 Dec 2016. It is approved in 3 of the 3 regulators tracked here.
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.
Nusinersen patents and exclusivity
Expiry tracker →Protections still running for marketed products containing Nusinersen, as listed by the regulators. Expired patents and exclusivities, and discontinued products, are not shown.
- 7 listed patents; the last listed expires 4 Mar 2036 (US 12013403).
- Latest patent on the drug substance itself: 23 Dec 2030 (US 8361977).
- Exclusivity NS: New strength, until 27 Mar 2029.
- Spinraza for treatment of 5q spinal muscular atrophy: until 1 Jun 2029 (includes 24 months' paediatric extension). Union Register
Reproduced from the FDA Orange Book and Purple Book (checked 5 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 Nusinersen
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 Nusinersen works
All drug targets →- SMN2 pre-mRNA positive modulator Positive modulatorTarget: SMN2 survival of motor neuron 2, centromericChEMBL only
- Proximal spinal muscular atrophyPhase 3
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL3301572, CC BY-SA 3.0. Targets checked against the IUPHAR/BPS Guide to PHARMACOLOGY (2026.3), ligand 9416, 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 Nusinersen
All NICE appraisals →NICE, which decides whether the NHS in England should fund new medicines, has published 2 technology appraisals of Nusinersen. 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 |
| Nusinersen for treating spinal muscular atrophy NICE TA588 | Optimised | 24 Jul 2019 | 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 Nusinersen
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- IEIreland: 3 products on the HPRA listEach product record links to the HPRA, which publishes the leaflet and SmPC.
- EUEuropean Union: SpinrazaEMA 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 Nusinersen
- ATC code
- M09AX07
- ChEMBL
- CHEMBL3301572
- DrugBank
- DB13161
- PubChem CID
- 168313233
- CAS number
- 1258984-36-9
- Wikidata
- Q25105466
- Wikipedia
- Nusinersen
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 Spinraza is used for
SPINRAZA is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. SPINRAZA is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients ( 1 )
How it works
12.1 Mechanism of Action SPINRAZA is an antisense oligonucleotide (ASO) designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Using in vitro assays and studies in transgenic animal models of SMA, SPINRAZA was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein.
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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 4 Oct 2026. How every register is built: methodology · fixes we have made: corrections log.