Tofersen

Phase 3

Phase 3

Development Phase

Upcoming Catalysts

Historical Events

Regulatory Approvals

No catalyst events have been recorded for this drug yet.

Competitive Landscape

0 competitors in ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation

Drug Information

Target Indication

ALS Caused by Superoxide Dismutase 1 (SOD1) Mutation

Clinical Trial

Last updated: 10/17/2026

Patent & Exclusivity

No patent data available. Pre-approval drugs may not have Orange Book listings.

Drug Science

Mechanism of action & safety profile

Developer

IONS

Ionis Pharmaceuticals, Inc.

Related Pipeline

Angelman Syndrome

Familial Chylomicronemia Syndrome

Familial Chylomicronemia Syndrome

FAP

Amyotrophic Lateral Sclerosis

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