CurrentUnitedHealthcarePolicy CSLA2025D00123C

Qalsody (Tofersen) Medical Benefit Drug Policy

UnitedHealthcare Louisiana-only medical benefit drug policy governing coverage criteria, authorization durations, and applicable billing codes for Qalsody (tofersen) for treatment of amyotrophic lateral sclerosis (ALS) in patients with SOD1 gene mutation.

Policy Summary

PayerUnitedHealthcare

PolicyQalsody (Tofersen) Medical Benefit Drug Policy

Policy CodePolicy CSLA2025D00123C

Change TypeRevised coverage criteriaauthorization duration updated

Effective DateAug 1, 2025

Next Review Date

Key ActionProvider must submit medical records to support ALS diagnosis and confirm SOD1 mutation and attest baseline functional ability prior to initiating Qalsody.

SourceLink

POLICY UPDATE CHANGES

Revised coverage criteria; removed criterion requiring the member is not dependent on tracheostomy.

Replaced language indicating 'authorization will be for no more than 6 months' with 'authorization will be for no more than 12 months'.

Updated Clinical Evidence and References sections to reflect the most current information.

1HCPCS drug code listed

1Diagnosis code listed

12 monthsMaximum authorization duration

Coverage Summary

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Initial Therapy

Covered when ALL of the following are met:

ALL of the following

Submission of medical records to support the diagnosis of ALS (e.g., chart notes, previous medical history, diagnostic testing including: imaging, nerve conduction studies, laboratory values).
Submission of medical records confirming mutation in the superoxide dismutase 1 (SOD1) gene (genetic test results).
Provider attestation that the patient's baseline functional ability has been documented prior to initiating treatment (e.g., speech, walking, climbing stairs, ALSFRS-R or equivalent).
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.
Initial authorization will be for no more than 12 months.

Documentation Required

Submit supporting medical records and a baseline functional attestation at initiation. Expected documents include: chart notes documenting ALS signs/symptoms and prior medical history; diagnostic testing (imaging, nerve conduction studies, pulmonary function tests, laboratory values); genetic test results confirming SOD1 mutation; and a provider attestation of baseline functional ability (e.g., ALSFRS-R or narrative documenting speech, walking, climbing stairs).

Chart notes documenting diagnosis and prior medical history
Diagnostic testing results (imaging, nerve conduction studies, pulmonary function tests, laboratory values)
Genetic test results confirming SOD1 mutation
Baseline functional attestation (ALSFRS-R score or documented functional abilities)

Prior Authorization

Prior Authorization Required

Qalsody must be prescribed by, or in consultation with, a neurologist with expertise in ALS. Prior authorization is required for J1304 (Injection, tofersen, 1 mg). Initial prior authorization will be granted for up to 12 months. For continuation, provider must submit response/continuation attestation documenting slowed disease progression from baseline.

HCPCS: J1304 — Injection, tofersen, 1 mg
Initial prior authorization duration: up to 12 months
Provider continuation requirement: attestation of clinical response/slowed disease progression from baseline

Continuation of Therapy

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
Qalsody dosing for ALS is in accordance with the United States Food and Drug Administration approved labeling
Authorization will be for no more than 12 months
Authorization will be for no more than 12 months

Documentation Required

Continuation Documentation

For continuation requests, the provider must attest that the patient has slowed disease progression compared to baseline to justify ongoing Qalsody therapy. Acceptable evidence includes documented changes in validated functional measures or clinical progress notes.

Change in ALSFRS-R score from baseline
Clinical progress notes documenting slowed progression
Objective measures noted in chart (e.g., SVC, hand-held dynamometry)

Prior Authorization

Prior Authorization / Renewal

Continuation (renewal) requests require prior authorization. Qalsody must be prescribed by, or in consultation with, a neurologist with expertise in ALS; authorization for continuation will be for no more than 12 months. Include J1304 when applicable.

J1304

Provider Actions & Requirements

Documentation Required

Documentation Needed

Submit supporting medical records and a baseline functional attestation prior to initiating Qalsody. Documentation must confirm ALS diagnosis, SOD1 mutation status, and baseline functional status.

Chart notes and previous medical history
Diagnostic testing (e.g., imaging, nerve conduction studies, laboratory values)
Genetic test results confirming SOD1 mutation
Document baseline functional status (e.g., ALSFRS-R, speech, mobility)

Prior Authorization

Prior Authorization / Specialist Requirement

Qalsody must be prescribed by, or in consultation with, a neurologist with expertise in ALS. Prior authorization is required for both initial and continuation requests and will be for no more than 12 months.

J1304

Note

Continuation Attestation

For renewals, the provider must attest that the patient has slowed disease progression from baseline to justify ongoing therapy with Qalsody.

Clinical Evidence

108Patients randomized (Study 1 Part C)

28 weeksRCT duration (primary study)

12 monthsMaximum authorization duration

1HCPCS drug code listed (J1304)

Key randomized trial: Study 1 Part C (NCT02623699) was a 28-week randomized, double-blind, placebo-controlled trial (108 patients randomized 2:1) in patients with SOD1-ALS. The prespecified primary analysis in the modified intent-to-treat (mITT) population did not show a statistically significant benefit on the primary clinical endpoint (ALSFRS-R change at Week 28). However, secondary biomarker endpoints — reductions in plasma neurofilament light chain (NfL) and CSF SOD1 protein — were nominally statistically significant.

Open-label extension: After Study 1, 95 participants entered an open-label extension. At 52 weeks, exploratory analyses suggested reductions in NfL among delayed-start patients who initiated tofersen and non-multiplicity-adjusted differences favoring earlier initiation for ALSFRS-R and other clinical measures; trends for reduced risk of death or permanent ventilation were observed but were not statistically significant. Lumbar puncture–related adverse events were common and neurologic serious adverse events occurred in a minority of recipients.

Guideline and regulatory context: The FDA granted accelerated approval of Qalsody based on the reduction in plasma NfL with continued approval contingent on confirmatory clinical benefit. The 2024 European Academy of Neurology (EAN) guideline recommends offering tofersen as first-line treatment for progressive ALS caused by pathogenic SOD1 mutations while discussing the potential for serious adverse events.

Term	Definition
SOD1	Superoxide dismutase 1 gene; pathogenic mutations associated with familial ALS.
ALSFRS-R	Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised, a measure of ALS functional status.

Revision History

2025-08-01policy_revisionLatest

Material: Removed prior exclusion specific to tracheostomy dependence — revised coverage criteria no longer require member to be not dependent on tracheostomy.

2025-08-01policy_revisionLatest

Material: Authorization duration changed from no more than 6 months to no more than 12 months for initial and continuation therapy.

2025-08-01supporting_information_updateLatest

Non-material: Updated Clinical Evidence and References sections to reflect the most current information.

Policy Summary

PayerUnitedHealthcare

PolicyQalsody (Tofersen) Medical Benefit Drug Policy

Policy CodePolicy CSLA2025D00123C

Change TypeRevised coverage criteriaauthorization duration updated

Effective DateAug 1, 2025

Next Review Date

Key ActionProvider must submit medical records to support ALS diagnosis and confirm SOD1 mutation and attest baseline functional ability prior to initiating Qalsody.

SourceLink

On This Page

OpenPayer

Qalsody (Tofersen) Medical Benefit Drug Policy

Coverage Summary

Initial Therapy

Continuation of Therapy

Applicable Codes

Provider Actions & Requirements

Clinical Evidence

Background & Definitions

Revision History