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Qalsody (TOFERSEN)
Qalsody works by binding to the messenger RNA (mRNA) of the Superoxide dismutase 1 (SOD1) gene, preventing its expression and reducing the production of the toxic SOD1 protein associated with ALS.
At a glance
| Generic name | TOFERSEN |
|---|---|
| Sponsor | Biogen Ma |
| Drug class | Antisense Oligonucleotide [EPC] |
| Target | Superoxide dismutase 1 (SOD1) mRNA |
| Modality | Oligonucleotide |
| Therapeutic area | Rare Disease |
| Phase | FDA-approved |
| First approval | 2023 |
| Annual revenue | 200 |
Mechanism of action
. Mechanism of Action. Tofersen is an antisense oligonucleotide that causes degradation of SOD1 mRNA through binding to SOD1 mRNA, which results in reduction of SOD1 protein synthesis.
Approved indications
- Amyotrophic lateral sclerosis
Common side effects
- Pain
- Fatigue
- Arthralgia
- CSF white blood cell increased
- Myalgia
- CSF protein increased
- Musculoskeletal stiffness
- Neuralgia
- Pleocytosis
- Pyrexia
- Aseptic meningitis
- Myelitis
Key clinical trials
- A Study of BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation (PHASE3)
- 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)
- Tofersen in Non-SOD1 ALS (PHASE2)
- A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic Lateral Sclerosis (ALS) (PHASE4)
- Long-Term Evaluation of BIIB067 (Tofersen) (PHASE3)
- Registry Study of Assistive Devices, Medicines and Healthcare Measures in ALS, SMA and Other Neurological Diseases.
- Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis
- Rehabilitation in SOD1 ALS Treated With Tofersen
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
| SEC EDGAR | Revenue + earnings |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Qalsody CI brief — competitive landscape report
- Qalsody updates RSS · CI watch RSS
- Biogen Ma portfolio CI