CurrentUnitedHealthcarePolicy 2026D0123F

Qalsody® (Tofersen) – Commercial and Individual Exchange Medical Benefit Drug Policy

Defines UnitedHealthcare Commercial and Individual Exchange medical benefit coverage criteria, documentation requirements, prescribing provider qualifications, dosing alignment with FDA labeling, authorization durations, applicable codes, and supporting evidence for Qalsody (tofersen) for treatment of amyotrophic lateral sclerosis (ALS) in patients with SOD1 mutation.

Policy Summary

PayerUnitedHealthcare

PolicyQalsody® (Tofersen) – Commercial and Individual Exchange Medical Benefit Drug Policy

Policy CodePolicy 2026D0123F

Change TypeTemplate update; references refreshed

Effective DateMay 1, 2026

Next Review Date

Key ActionProvider must submit medical records to support ALS diagnosis and confirm SOD1 mutation, document baseline functional ability, and ensure Qalsody is prescribed by or in consultation with an ALS-experienced neurologist with dosing per FDA labeling.

SourceLink

POLICY UPDATE CHANGES

Transferred content to shared policy template that applies to both UnitedHealthcare Commercial and Individual Exchange benefit plans and added Application section.

1Indication covered

12 moMax auth (initial/renewal)

J1304Primary billing code

Coverage Summary

Scope: Defines UnitedHealthcare Commercial and Individual Exchange medical benefit coverage criteria, documentation requirements, prescribing provider qualifications, dosing alignment with FDA labeling, authorization durations, applicable codes, and supporting evidence for Qalsody (tofersen) for treatment of amyotrophic lateral sclerosis (ALS) in patients with an SOD1 mutation. Coverage stance: covered_with_criteria. Effective/Last review date: 2026-05-01.

Initial therapy

Covered when ALL of the following are met:

ALL of the following

Submission of medical records (e.g., chart notes, previous medical history, diagnostic testing including: imaging, nerve conduction studies, laboratory values) to support the diagnosis of ALS
Submission of medical records confirming mutation in the superoxide dismutase 1 (SOD1) gene
Provider attestation that the patient's baseline functional ability has been documented prior to initiating treatment (e.g., speech, walking, climbing stairs, etc.)
Patient is not dependent on invasive ventilation
Qalsody is prescribed by, or in consultation with, a neurologist with expertise in the diagnosis of ALS
Qalsody dosing for ALS is in accordance with the United States Food and Drug Administration approved labeling
Initial authorization will be for no more than 12 months

Continuation of therapy

Covered when ALL of the following are met:

ALL of the following

Diagnosis of ALS
Patient is currently receiving Qalsody therapy
Provider attestation that the patient has slowed disease progression from baseline
Patient is not dependent on invasive ventilation
Qalsody is prescribed by, or in consultation with, a neurologist with expertise in the diagnosis of ALS

Prior Authorization & Documentation Requirements

Prior Authorization

Prescriber specialty and dosing alignment

Qalsody must be prescribed by, or in consultation with, a neurologist experienced in ALS and dosing must follow FDA-approved labeling. Initial and continuation authorizations are limited to no more than 12 months.

J1304

Documentation Required

Submit medical records confirming ALS diagnosis and SOD1 mutation

Provider must submit medical records including chart notes, diagnostic testing (imaging, nerve conduction studies, labs) to support the diagnosis of ALS and documentation confirming SOD1 gene mutation.

Documentation Required

Baseline functional assessment attestation

Provider must attest that the patient's baseline functional ability (e.g., speech, walking, climbing stairs) has been documented prior to initiating treatment.

Documentation Required

Continuation attestation of slowed disease progression

For renewal, provider must attest that the patient has slowed disease progression from baseline while on therapy.

Initial and continuation authorizations are limited to no more than 12 months per the policy; initial authorization for Qalsody will be for no more than 12 months, and continuation/renewal authorizations are likewise limited to no more than 12 months. Operational notes require submission of supporting medical records, SOD1 mutation confirmation, baseline functional assessment attestation, and that prescribing be by or in consultation with a neurologist experienced in ALS and dosing consistent with FDA labeling.

Clinical Evidence Summary

Primary randomized trial: In Study 1 Part C (NCT02623699) 108 patients were randomized 2:1 to tofersen versus placebo; the prespecified primary endpoint (change in ALSFRS-R at 28 weeks in the mITT population) was not statistically significant, although plasma NfL and CSF SOD1 protein reductions were nominally significant.

Open-label extension findings: An interim analysis at 52 weeks of the open-label extension showed a difference in ALSFRS-R change favoring early-start over delayed-start of 3.5 points (95% CI 0.4 to 6.7), with reductions in NfL observed in patients initiating tofersen. These exploratory/open-label results should be interpreted with caution.

Safety signal: Lumbar puncture–related adverse events were common, and neurologic serious adverse events occurred in approximately 7% of tofersen recipients. The FDA approved Qalsody under accelerated approval based on reduction in plasma neurofilament light chain, with continued approval contingent on verification of clinical benefit.

Background & Definitions

Qalsody (tofersen) is an antisense oligonucleotide that binds SOD1 mRNA and causes degradation of SOD1 mRNA, reducing SOD1 protein synthesis. It is indicated for treatment of ALS in adults with an SOD1 gene mutation and was approved under the FDA’s accelerated approval pathway based on reduction in plasma neurofilament light chain; continued approval may be contingent on confirmatory trials verifying clinical benefit. Amyotrophic lateral sclerosis (ALS) is a rapidly progressive, fatal motor neuron disease characterized by degeneration of motor neurons, progressive loss of voluntary muscle control, respiratory failure risk, and typical survival of about 3 to 5 years from symptom onset; familial ALS constitutes ~10% of cases and SOD1 mutations account for a subset of familial disease.

Definitions and Policy Identifiers

termSOD1-ALS

definitionAmyotrophic lateral sclerosis caused by pathogenic mutations in the superoxide dismutase 1 (SOD1) gene.

Policy number2026D0123F

Effective date2026-05-01

Last review2026-05-01

StatusCURRENT

Policy Summary

PayerUnitedHealthcare

PolicyQalsody® (Tofersen) – Commercial and Individual Exchange Medical Benefit Drug Policy

Policy CodePolicy 2026D0123F

Change TypeTemplate update; references refreshed

Effective DateMay 1, 2026

Next Review Date

SourceLink

On This Page

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Qalsody® (Tofersen) – Commercial and Individual Exchange Medical Benefit Drug Policy

Coverage Summary

Prior Authorization & Documentation Requirements

Applicable Codes

Clinical Evidence Summary

Background & Definitions

Revision History