Amyotrophic lateral sclerosis with a mutation in the superoxide dismutase 1 gene

Active Ingredient: Tofersen

Indication for Tofersen

Population group: only adults (18 years old or older)
Therapeutic intent: Curative procedure

Tofersen is indicated for the treatment of adults with amyotrophic lateral sclerosis (ALS), associated with a mutation in the superoxide dismutase 1 (SOD1) gene.

For this indication, competent medicine agencies globally authorize below treatments:

3 loading doses of 100 mg once every 14 days and thereafter 100 mg every 28 days

For:

Dosage regimens

Intraspinal, 100 milligrams tofersen, once every 14 days, 3 doses in total. Afterwards, intraspinal, 100 milligrams tofersen, once every 28 days.

Detailed description

The recommended dose is 100 mg of tofersen per treatment.

Tofersen treatment should be initiated with 3 loading doses administered at 14-day intervals.

A maintenance dose should be administered once every 28 days thereafter.

Missed or delayed doses

If the second loading dose is delayed or missed, tofersen should be administered as soon as possible, and the third loading dose should be administered 14 days later.

If the third loading dose is delayed or missed, tofersen should be administered as soon as possible, and the first maintenance dose should be administered 28 days later.

If a maintenance dose is delayed or missed, tofersen should be administered as soon as possible. Subsequent maintenance doses should be administered every 28 days from the last dose.

Duration of treatment

The need for continuation of therapy should be reviewed regularly and considered on an individual basis depending on the patient’s clinical presentation and response to the therapy.

Elderly

Experience with the use of tofersen in the elderly is limited. However, from the clinical data available, the efficacy and safety of tofersen are expected to be similar to that of other age groups studied. There is no evidence for special dose considerations based on age when tofersen is administered.

Dosage considerations

Tofersen is for intrathecal use by lumbar puncture.

Procedural preparation instruction:

  • If indicated by the clinical condition of the patient, sedation can be considered.
  • If indicated by the clinical condition of the patient, imaging to guide intrathecal administration of tofersen can be considered.
  • Patients should be evaluated prior to and after intrathecal injection for the presence of potential conditions related to lumbar puncture to avoid serious procedural complications.

Following injection, standard post-lumbar-puncture care is recommended.

Active ingredient

Tofersen

SOD1-ALS is a primarily autosomal-dominant disorder affecting approximately 2% of the ALS population. Mutations in the SOD1 gene lead to accumulation of a toxic form of SOD1 protein. Tofersen is an antisense oligonucleotide (ASO) that is complementary to a portion of the 3′ untranslated region (3′UTR) of the mRNA for human SOD1 and binds to the mRNA by Watson-Crick base pairing (hybridisation). This hybridisation of tofersen to the cognate mRNA results in RNase-Hmediated degradation of the mRNA for SOD1, which reduces the amount of SOD1 protein synthesis.

Read more about Tofersen

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