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Drug

Tofersen

Approved · EMA
Late-stage development

Also known as Antisense Oligonucleotide Inhibitor Of The Expression Of Superoxide Dismutase 1 Gene, DNA, D((2'-O-(2-METHOXYETHYL))M5RC-SP-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))RG-SP-(2'-O-(2-METHOXYETHYL))RG-(2'-O-(2-METHOXYETHYL))RA-SP-T-SP-A-SP-M5C-SP-A-SP-T-SP-T-SP-T-SP-M5C-SP-T-SP-A-SP-(2'-O-(2-METHOXYETHYL))M5RC-(2'-O-(2-METHOXYETHYL))R, IONIS SOD1Rx, Qalsody.

RxNorm2634995UNII2NU6F9601K
3
Active clinical trials
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Study to Evaluate the Biological Effect of Tofersen in Adults With Amyotrophic Lateral Sclerosis Without Mutations in SOD1

Clinical trial2026-06-25Results expected Q1 2027 · ClinicalTrials.gov

A Phase 3 Randomized, Placebo-Controlled Trial With a Longitudinal Natural History Run-In and Open-Label Extension to Evaluate BIIB067 Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation

Clinical trial2026-03-30Results expected Q3 2027 · ClinicalTrials.gov

A Multicenter, Open-Label, Postmarketing Surveillance Study of Tofersen (BIIB067) in Adults With Amyotrophic Lateral Sclerosis (ALS) Associated With a Mutation in the Superoxide Dismutase 1 (SOD1) Gene in China

Clinical trial2026-01-20Results expected Q4 2027 · ClinicalTrials.gov

An Extension Study to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB067 Administered to Previously Treated Adults With Amyotrophic Lateral Sclerosis Caused by Superoxide Dismutase 1 Mutation

Clinical trial2025-08-29Results posted · ClinicalTrials.gov

Approval: Qalsody (EMA)

Regulatory2024-05-29EMA

A Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB067 Administered to Adult Subjects With Amyotrophic Lateral Sclerosis and Confirmed Superoxide Dismutase 1 Mutation

Clinical trial2023-07-28Results posted · ClinicalTrials.gov

A Phase 1, Safety, Tolerability, and Distribution Study of a Microdose of Radiolabeled BIIB067 Co-administered With BIIB067 to Healthy Adults

Clinical trial2021-07-10Completed · ClinicalTrials.gov

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Tofersen
Aliases & brands
Antisense Oligonucleotide Inhibitor Of The Expression Of Superoxide Dismutase 1 GeneDNA, D((2'-O-(2-METHOXYETHYL))M5RC-SP-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))RG-SP-(2'-O-(2-METHOXYETHYL))RG-(2'-O-(2-METHOXYETHYL))RA-SP-T-SP-A-SP-M5C-SP-A-SP-T-SP-T-SP-T-SP-M5C-SP-T-SP-A-SP-(2'-O-(2-METHOXYETHYL))M5RC-(2'-O-(2-METHOXYETHYL))RIONIS SOD1RxQalsody
RxNorm CUI
2634995
UNII
2NU6F9601K
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2024-05-29
Latest approval
2024-05-29
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2024-05-29Approval
Approval: Qalsody (EMA)

Indication: Qalsody is indicated for the treatment of adults with amyotrophic lateral sclerosis (ALS), associated with a mutation in the superoxide dismutase 1 (SOD1) gene.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

6 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
6
registered trials across all phases
LATEST COMPLETION 2024
3
Active studies
2
Recruiting
4
Late-stage (III+)
3
Completed
PHASE DISTRIBUTIONn = 6
Phase 11Phase 21Phase 33Phase 41

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

A Multicenter, Open-Label, Postmarketing Surveillance Study of Tofersen (BIIB067) in Adults With Amyotrophic Lateral Sclerosis (ALS) Associated With a Mutation in the Superoxide Dismutase 1 (SOD1) Gene in China

RecruitingPH IVBiogenn=122027

A Phase 3 Randomized, Placebo-Controlled Trial With a Longitudinal Natural History Run-In and Open-Label Extension to Evaluate BIIB067 Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation

Active, not recruitingPH IIIBiogenn=1582028

An Extension Study to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB067 Administered to Previously Treated Adults With Amyotrophic Lateral Sclerosis Caused by Superoxide Dismutase 1 Mutation

CompletedPH IIIBiogenn=1392024

A Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB067 Administered to Adult Subjects With Amyotrophic Lateral Sclerosis and Confirmed Superoxide Dismutase 1 Mutation

CompletedPH IIIBiogenn=1762021
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.