How is treatment with Tofersen administered?

Tofersen is administered via a lumbar puncture using a fine needle into the spinal canal (“intrathecal”) so that the active ingredient can reach the central nervous system (CNS). At the start of treatment, a “loading” regimen of three doses, each administered 14 days apart, is used to establish the therapy. Thereafter, a maintenance dose is administered every 28 days.

Treatment is provided on an outpatient basis, at a day clinic, or during a short inpatient stay. The lumbar puncture procedure takes less than an hour. The preparations, assessments, and measurements associated with the treatment can take several hours in total. Organizational arrangements may vary significantly among different treatment centers.