Qalsody®

Drug - Qalsody® (tofersen) [Biogen Inc.]

March 2025

Therapeutic Area - ALS Agents

Initial approval criteria

Patient is ≥ 18 years of age; AND
Patient has a baseline measure of plasma neurofilament light chain (NfL); AND
Patient has a diagnosis of clinically definite or probable amyotrophic lateral sclerosis (ALS) based on revised El Escorial criteria or Awaji criteria; AND
Patient has the presence of a superoxide dismutase 1 (SOD1) gene mutation; AND
Patient has a slow vital capacity (%SVC) ≥ 65%; AND
Baseline documentation of retained functionality for most activities of daily living (i.e., score of ≥ 2 on each item of the ALS Functional Rating Scale – Revised [ALSFRS-R]) has been obtained
Initial approval is for 6 months

Renewal criteria

Patient must continue to meet the above criteria; AND
Patient has not experienced any unacceptable toxicity from treatment (e.g., serious myelitis and radiculitis, papilledema and elevated cranial pressure, aseptic meningitis); AND
Patient must have stabilization OR improvement in plasma NfL compared to baseline; AND
Patient has responded to therapy compared to pretreatment baseline with disease stability or mild progression indicating a slowing of decline on the ALSFRS-R (patient has not experienced rapid disease progression while on therapy); AND
Patient does not have a cumulative ALSFRS-R score ≤ 3
Renewal approval is for 6 months

Quantity limits

1 vial (100 mg/15 mL) every 14 days for 3 doses; then 1 vial every 28 days

Billing for Qalsody

Qalsody must be billed as a medical claim.

Background Information

Qalsody is an antisense oligonucleotide indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. This indication is approved under accelerated approval based on reduction in plasma neurofilament light chain observed in patients treated with Qalsody. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).

Questions

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