Qalsody

(Tofersen)

ApprovedRNA therapy

Qalsody is a rna therapy that works as a SOD1 Antisense Oligonucleotide.[2] It is being used across 2 indications, with its most advanced program in Approved.[1]

Qalsody is one of 40 aso programs targeting Amyotrophic Lateral Sclerosis. It is currently in Approved.

Indications

Total Trials

Active Trials

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

Amyotrophic Lateral SclerosisActive, not recruiting

ALS (amyotrophic Lateral Sclerosis)Recruiting

Amyotrophic Lateral SclerosisApproved

Active, not recruiting

ALS (amyotrophic Lateral Sclerosis)Phase 2

Recruiting

Competition

Competitive Landscape

55 competitors in Amyotrophic Lateral Sclerosis

#DrugCompanyPhaseTrials

Vimpat

UCB

Approved

—2

Nourianz

Kyowa Kirin Co., Ltd.

Enablex

1 4

Bosulif

PPrism Pharmaceuticals

Approved

1 5

Rocephin

CConrado Roberto Hoffmann Filho

Approved

—6

Transderm Scop

BBaxter Healthcare Corporation

Mirapex

Avodart

Rapamune

PPrism Pharmaceuticals

Approved

—10

Precedex

MMcNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.

Approved

—

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q2 2027

ApprovedActive, not recruiting

Amyotrophic Lateral Sclerosis

NCT07223723

Q2 2028

Phase 2Recruiting

ALS (amyotrophic Lateral Sclerosis)

NCT07294144

Phase 3Active, not recruiting

Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation

NCT04856982

Q4 2033

Not yet recruiting

Amyotrophic Lateral Sclerosis

NCT07259980

Clinical

Trials by Indication[1]

Amyotrophic Lateral Sclerosis3 trials

An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)

Completed· Biogen logo

Biogen·Phase 3·NCT02623699·176 enrolled

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)

Not yet recruiting· Biogen logo

Biogen··NCT07259980·125 enrolled

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

Active, not recruiting· Biogen logo

Biogen·Approved·NCT07223723·12 enrolled

Adult1 trial

A Study to Evaluate Safety, Tolerability, and Distribution of a Microdose of Radiolabeled BIIB067 (99mTc-MAG3-BIIB067) Co-administered With BIIB067 (Tofersen) to Healthy Adults

Completed· Biogen logo

Biogen·Phase 1·NCT03764488·8 enrolled

ART1 trial

Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis

Approved for marketing· Biogen logo

Biogen··NCT04972487

Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation1 trial

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 recruiting· Eli Lilly logo

Eli Lilly·Phase 3·NCT04856982·158 enrolled

ALS (Amyotrophic Lateral Sclerosis)1 trial

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

Recruiting·WWashington University School of Medicine·Phase 2·NCT07294144·30 enrolled

1 more indication

Unspecified1 trial

Long-Term Evaluation of BIIB067 (Tofersen)

Completed· Biogen logo

Biogen·Phase 3·NCT03070119·139 enrolled

Regulatory

FDA Approval Timeline[4]

Apr 25, 2023

QALSODY

NDA NDA215887

Apr 25, 2023

QALSODY

NDA NDA215887

Sources

References

[1]
ClinicalTrials.gov — 8 trial records for Tofersen. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Tofersen compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Tofersen. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA215887). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Qalsody. U.S. Food & Drug Administration.
[6]
PubMed — 10 publications indexed for Tofersen. U.S. National Library of Medicine.
[7]
SEC EDGAR — Biogen company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: SOD1 Antisense Oligonucleotide[2]
Modality: RNA therapy[2]
Highest Phase: Approved[1]
Company: Biogen

Patent Landscape[5]

Patent Cliff

Apr 1, 2035

Earliest patent expiration — generic entry possible after this date

Patent #Expires

10968453Apr 1, 2035

10669546Apr 1, 2035

10385341Apr 1, 2035

Key Publications[6]

Artificial Intelligence Decision-Making in Multidisciplinary Tumor Boards for Head and Neck Cancer: A Systematic Review.

J Otolaryngol Head Neck SurgDec 1, 2026DOI: 10.1177/19160216261451777

Mature Adipocyte-Derived Extracellular Vesicles Activate Hepatic Stellate Cells and Promote Liver Fibrosis via Inflammation.

Liver IntNov 1, 2026DOI: 10.1111/liv.70914

From Mitochondrial Targeting to Mechanistic Dependency in Nanoparticle Therapy for Ulcerative Colitis.

J Gastroenterol HepatolOct 13, 2026DOI: 10.1111/jgh.70809

Efficacy of minocycline-containing bismuth quadruple therapy for Helicobacter pylori infection: a single-center retrospective study.

Expert Rev Clin PharmacolOct 13, 2026DOI: 10.1080/17512433.2026.2749579

The Effects of GLP-1RA-Based and SGLT2 Inhibitor Therapy on Perirenal Adipose Tissue in CKD Patients.

Diabetes Obes MetabOct 13, 2026DOI: 10.1111/dom.71441

Bibliometric analysis of the research trends of thalassemia in the past decade.

Expert Rev HematolOct 13, 2026DOI: 10.1080/17474086.2026.2748721

Single-NIR Triggered Triple-Modal Therapy: Synergistic PTT/PDT and Ultrafast Ag(II) Release via a Van Der Waals Silver Porphyrin/Graphene-TiO(2) Heterojunction for MRSA-Infected Wounds.

SmallOct 13, 2026DOI: 10.1002/smll.76222

SGLT2 Inhibitors Reduce Heart Failure Hospitalization, Cardiovascular Death and Kidney Outcomes in Type 2 Diabetes: A Meta-Analysis of Randomized Controlled Trials.

Diabetes Obes MetabOct 13, 2026DOI: 10.1111/dom.71411

Ketamine administered biomedically or as psychedelic-assisted therapy in treatment-resistant depression: Differing efficacy based on depressive subtype or presence of childhood trauma?

Australas PsychiatryOct 13, 2026DOI: 10.1177/10398562261494440

Soluble Indicators of Endothelial Injury and Activation Emerge During Acute HIV Infection and Persist Despite Rapid Antiretroviral Therapy Initiation.

J Infect DisOct 13, 2026DOI: 10.1093/infdis/jiag495

Qalsody

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q2 2027

Q2 2028

Q4 2033

Trials by Indication[1]

Amyotrophic Lateral Sclerosis3 trials

Adult1 trial

ART1 trial

Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation1 trial

ALS (Amyotrophic Lateral Sclerosis)1 trial

Unspecified1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

Key Publications[6]

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