.Pharmacolibrary.Drugs.N_NervousSystem.N07X_OtherNervousSystemDrugs.N07XX22_Tofersen.Tofersen

Information

name:Tofersen
ATC code:N07XX22
route:intrathecal
n-compartments1

Tofersen is an antisense oligonucleotide designed to reduce the production of superoxide dismutase 1 (SOD1) protein. It is indicated for the treatment of amyotrophic lateral sclerosis (ALS) in patients with SOD1 mutations. Tofersen was granted accelerated approval by the FDA in 2023 for use in this patient population.

Pharmacokinetics

Pharmacokinetic parameters reported in adults with SOD1-ALS following repeated intrathecal administration.

References

    Revisions

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