Tofersen clinical trials
Tofersen is named as an intervention in 3 current clinical trials in our USA, UK, Germany, France, Spain, Italy, Belgium, Sweden, Poland, Canada, Brazil, Japan, South Korea, Australia and China registers. 1 is recruiting patients now. The most common phase is Phase 3. The lead sponsor is Biogen. The most-studied condition is Amyotrophic Lateral Sclerosis.
Where Tofersen is approved
All regulators →Of the three regulators tracked here, the first to approve Tofersen was the US, on 25 Apr 2023. 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.
Tofersen patents and exclusivity
Expiry tracker →Protections still running for marketed products containing Tofersen, as listed by the regulators. Expired patents and exclusivities, and discontinued products, are not shown.
- 3 listed patents; the last listed expires 1 Apr 2035 (US 10968453).
- Latest patent on the drug substance itself: 1 Apr 2035 (US 10385341).
- Exclusivity ODE-432: Orphan drug exclusivity: treatment of amyotrophic lateral sclerosis (als) in adults who have a mutation in the superoxide dismutase 1 (sod1…, until 25 Apr 2030.
- Exclusivity NCE: New chemical entity, until 25 Apr 2028.
- Qalsody for treatment of amyotrophic lateral sclerosis: until 3 Jun 2034. Union Register
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 Tofersen
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 Tofersen works
All drug targets →- SOD1 mRNA antisense inhibitor Antisense inhibitorTarget: SOD1 superoxide dismutase 1ChEMBL only
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL3833346, CC BY-SA 3.0. Targets checked against the IUPHAR/BPS Guide to PHARMACOLOGY (2026.3.1), ligand 13536, CC BY-SA 4.0. Trial phases are not approvals: approved uses are shown from the regulators' own records where we hold them.
Patient leaflets for Tofersen
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- IEIreland: 1 product on the HPRA listEach product record links to the HPRA, which publishes the leaflet and SmPC.
- EUEuropean Union: QalsodyEMA 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.
Trials by status
Trials by phase
Clinical trials of Tofersen
3 trials| Study | Phase | Status | Lead sponsor | Registers |
|---|---|---|---|---|
|
A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Participants With Superoxide Dismutase 1 (SOD-1) Amyotrophic Lateral Sclerosis (ALS)
NCT07259980 · Amyotrophic Lateral Sclerosis
|
Observational | Recruiting | Biogen | USA |
|
A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic Lateral Sclerosis (ALS)
NCT07223723 · ClinicalTrials.gov ↗ · Amyotrophic Lateral Sclerosis
|
Phase 4 | Active, closed | Biogen | China |
|
Study to Learn If Tofersen (BIIB067) Can Delay ALS Symptoms and About Its Safety in Adults With a SOD1 Gene Mutation Who Have Not Yet Developed ALS Symptoms
NCT04856982 · Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation
|
Phase 3 | Active, closed | Biogen | UK, USA, Germany, France, Spain, Italy, Belgium, Sweden, Poland, Canada, Brazil, Japan, South Korea, Australia |
Registers 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.
Tofersen 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)
1 single-substance product containing Tofersen on the HPRA medicines list.
- Qalsody 100 mg solution for injectionBiogen Netherlands B.V. · EU/1/23/1783/001
Supply shortages
No entry for Tofersen in the EMA, FDA or German BfArM shortage lists we track.
Conditions studied with Tofersen
2 conditionsSponsors
lead sponsor per trial| Lead sponsor | Trials |
|---|---|
| Biogen | 3 |
By country register
A trial with sites in more than one country appears in each of those registers.
Related compounds
Questions about Tofersen trials
How many clinical trials are testing Tofersen?
3 current trials in our USA, UK, Germany, France, Spain, Italy, Belgium, Sweden, Poland, Canada, Brazil, Japan, South Korea, Australia and China registers name Tofersen (also registered as BIIB067) as a drug or biological intervention, as of the registry sync on 9 November 2026. Completed and withdrawn studies are not counted.
Are Tofersen trials recruiting patients?
Yes. 1 of the 3 trials is recruiting: 1 with sites in USA. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.
Who sponsors Tofersen clinical trials?
The lead sponsor is Biogen. The number in brackets is how many trials each organisation leads.
Is Tofersen licensed in Ireland?
Yes. The HPRA medicines list includes 1 single-substance product containing Tofersen, for example Qalsody 100 mg solution for injection (Biogen Netherlands B.V.). A licence does not mean a product is currently marketed or reimbursed; see each product's record.
Is there a Tofersen shortage?
Not in the lists we track: Tofersen 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 Tofersen 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 Tofersen 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 Tofersen (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.