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tofersen
N07XX22
Forms & Strengths
- Intrathecal injection: 100 mg/15 mL (6.7 mg/mL) in a single-dose glass vial
Adult Dosing
- Initial: 100 mg (15 mL) intrathecally once every 14 days for 3 doses
- Maintenance: 100 mg (15 mL) intrathecally once every 28 days thereafter
Pediatric Dosing
- Safety and efficacy in pediatric patients have not been established
Indications
- Treatment of amyotrophic lateral sclerosis (ALS) in adults with a mutation in the superoxide dismutase 1 (SOD1) gene
Mechanism of Action
- Antisense oligonucleotide (ASO) designed to bind to SOD1 mRNA, promoting its degradation by ribonuclease H-mediated cleavage, thereby reducing synthesis of mutant SOD1 protein
Contraindications
- History of severe hypersensitivity reaction to tofersen or any of its excipients
Adverse Reactions
- Pain, musculoskeletal (23%)
- Fatigue (18%)
- Cerebrospinal fluid white blood cell increased (15%)
- Myalgia (13%)
- Pyrexia (12%)
- Procedural pain (11%)
- Headache (10%)
- Potential risks include myelitis, radiculitis, intracranial hypertension, and aseptic meningitis
Drug Interactions
- Formal clinical drug-drug interaction studies have not been performed
- Based on its clearance pathway, pharmacokinetic drug interactions with other medications are unlikely
Curated Content: Needs Vetting Before Put to Clinical Use