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Synonyms: BIIB-067 | BIIB067 | Qalsody®
tofersen is an approved drug (FDA (2023), EMA (2024))
Compound class:
Nucleic acid
Comment: Tofersen (BIIB067) is an antisense oligonucleotide that disrupts translation of superoxide dismutase 1 (SOD1). Tofersen was designed to treat cases of amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND) that are associated with expression of mutated SOD1. SOD1-ALS/MND accounts for ~2% of all cases.
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| No information available. |
Summary of Clinical Use  |
| Tofersen (Qalsody®) is approved to treat SOD1-ALS/MND. |
| Clinical Trials | |||||
| Clinical Trial ID | Title | Type | Source | Comment | References |
| NCT02623699 | An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS) | Phase 3 Interventional | Biogen | 1 | |
| NCT03070119 | Long-Term Evaluation of BIIB067 (Tofersen) | Phase 3 Interventional | Biogen | 1 | |
