CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Qalsody (tofersen) (Intrathecal) — Coverage Criteria

Medical coverage criteria, dosing, and authorization requirements for intrathecal Qalsody (tofersen) for patients with SOD1‑mutation ALS for Neighborhood Health Plan of Rhode Island members (Medicaid, Commercial, Medicare).

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyQalsody (tofersen) (Intrathecal) — Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material changes

Effective Date01/01/2024

Next Review Date06/11/2025

Key ActionSubmit prior authorization documenting age ≥18, confirmed SOD1 mutation, baseline plasma NfL, ALSFRS‑R item scores ≥2, and %SVC ≥65% for approval.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

6 monthsInitial length

100 mgPer-dose amount

3Loading doses

28 daysMaintenance interval

G12.21Covered Dx

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Initial Therapy — Covered when ALL of the following are met

Covered when ALL of the following are met

Eligibility and prescriber: Patient is at least 18 years of age; medication prescribed by a neurologist, neuromuscular specialist, or physician specializing in the treatment of ALS; baseline plasma neurofilament light chain (NfL) measured

Medicare members who received medication within past 365 days are not subject to step therapy requirements

Diagnosis and genetic requirement: Diagnosis of clinically definite or probable ALS based on El Escorial revised criteria or Awaji criteria; presence of a mutation in the SOD1 gene

Pulmonary function and functional status: Slow vital capacity (SVC) ≥ 65%; baseline documentation of retained functionality for most activities of daily living defined as a score of 2 or better on each individual ALSFRS-R item (minimum possible total 24)SVC >=65%; ALSFRS-R item score >=2 each

ALSFRS-R is a 12-item questionnaire; score of 2 on each item corresponds to minimum total of 24

Continuation / Renewal — Renewal provided when ALL of the following are met

Renewal provided when ALL of the following are met

Continued indication and toxicity: Patient continues to meet the indication-specific relevant criteria; absence of unacceptable toxicity from the drug (examples: serious myelitis/radiculitis, papilledema/elevated intracranial pressure, aseptic meningitis)

Biomarker and clinical response: Improvement in plasma NfL levels compared to baseline; patient has responded with disease stability or mild progression indicating slowing of decline on the ALSFRS-R (no rapid progression on therapy); patient does not have a cumulative ALSFRS-R score ≤ 3ALSFRS-R total >3

Cumulative score of 3 indicates loss/significant impairment in nine or more items

Requests for Qalsody (tofersen) are not authorized when the patient does not meet required baseline eligibility or assessment thresholds. Specifically, therapy is not authorized for patients who do not have a documented SOD1 gene mutation, who have a measured slow vital capacity (%SVC) <65%, who lack a documented baseline plasma neurofilament light chain (NfL) measurement, or whose baseline ALSFRS‑R item scores indicate insufficient retained function (any individual ALSFRS‑R item score <2) or a cumulative ALSFRS‑R total consistent with severe impairment (≤3).

Use of Qalsody outside the FDA‑approved population for adults with ALS due to a SOD1 mutation or without the policy’s required baseline and ongoing assessments is considered not medically necessary. The policy requires documentation of a clinically definite or probable ALS diagnosis (El Escorial revised or Awaji criteria), confirmation of a SOD1 mutation, baseline %SVC ≥65%, baseline ALSFRS‑R item scores of 2 or greater on each item, and a baseline plasma NfL level prior to initiating therapy. For continuation, the policy requires evidence of improved plasma NfL versus baseline and clinical stability or slowed decline on ALSFRS‑R, together with absence of unacceptable drug toxicity.

Billing and diagnosis codes

HCPCS CodeHCPCSCovered

J1304

Injection, tofersen, 1 mg

NDCNDC

64406-0109-xx

NDC for 100 mg/15 mL solution in single-dose glass vial (preservative free)

Covered Diagnosis CodesICD-10Covered

G12.21

Amyotrophic lateral sclerosis

inv-08: Slow vital capacity (%SVC) — threshold requirement

Threshold valueSlow vital capacity (SVC) >= 65%

Role in eligibilityRequired at baseline for initial approval — patient must have %SVC ≥65%

Renewal implicationRequests may be denied or therapy discontinued if %SVC <65% at baseline or shows unacceptable decline consistent with exclusion criteria

Source guidanceSpecified in initial coverage criteria and referenced in renewal criteria as part of indication-specific requirements

What providers must do

Prior Authorization

Prior Authorization Required

Prior authorization is required. Initial approval is granted for 6 months and may be renewed per renewal criteria.

Initial approval: 6 months
Max units/dosing details: HCPCS J1304 — 1 mg = 100 mg per billable unit (100 billable units = 100 mg)
Initial dosing schedule: 100 billable units (100 mg) every 14 days for 3 doses only; subsequent dosing: 100 billable units (100 mg) every 28 days thereafter

Step Therapy

Step therapy exception for prior users

Medicare members with documented prior use of this medication within the past 365 days are exempt from step therapy requirements.

Applies to Medicare members with prior receipt within 365 days

Documentation Required

Required documentation

Prescribers must submit documentation supporting the indication and baseline assessments. Required documentation includes age, baseline plasma neurofilament light chain (NfL), SOD1 mutation status, baseline ALSFRS‑R item scores, slow vital capacity (≥65%), and that the prescriber is a neurologist/neuromuscular specialist or physician specializing in ALS.

Patient is ≥ 18 years of age
Baseline plasma NfL value documented
Genetic testing documenting a pathogenic SOD1 mutation
Baseline ALS Functional Rating Scale—Revised (ALSFRS‑R) with item-level scores showing a score ≥2 on each individual item (minimum total 24)
Baseline slow vital capacity (SVC) documented and ≥ 65%
Prescribing clinician specialty documented (neurologist, neuromuscular specialist, or ALS specialist)

Denial Risk

Potential denial triggers

Coverage may be denied if the member does not meet diagnostic or baseline criteria, has unacceptable toxicity, or has advanced functional loss. Common denial triggers include age <18, no documented baseline NfL, absence of SOD1 mutation, SVC <65%, ALSFRS‑R item scores indicating insufficient retained function, or cumulative ALSFRS‑R score ≤3 at renewal.

Age under 18 years
No baseline plasma NfL documented
No documented SOD1 mutation
Slow vital capacity (SVC) < 65%
Baseline ALSFRS‑R item scores with any item <2 (insufficient retained function)
At renewal: cumulative ALSFRS‑R score ≤ 3 or evidence of rapid disease progression while on therapy
Evidence of unacceptable toxicity (e.g., serious myelitis/radiculitis, papilledema with elevated intracranial pressure, aseptic meningitis)

Background on Qalsody (tofersen)

Qalsody (tofersen) is an antisense oligonucleotide indicated for the treatment of adults with amyotrophic lateral sclerosis caused by mutations in the SOD1 gene. Regulatory approval was granted based on a reduction in plasma neurofilament light (NfL), a biomarker of axonal injury and neurodegeneration that the FDA considers reasonably likely to predict clinical benefit, despite the Phase 3 VALOR trial not meeting its primary ALSFRS‑R endpoint. In VALOR, patients receiving Qalsody had a substantially greater reduction in plasma NfL compared with placebo; common adverse events observed included pain, fatigue, arthralgia, increased CSF white blood cell count, and myalgia.

Definitions and clinical measures

inv-14: ALSFRS-R — description of the 12-item functional rating scale

DefinitionALSFRS-R is a 12-item questionnaire assessing functional disease progression across four domains: bulbar, fine motor, gross motor, and respiratory; each item scored 0–4 for a total possible score of 0–48

Scoring requirement for initial approvalPolicy requires a score of 2 or better on each individual ALSFRS-R item (minimum possible total 24) for initial coverage

Use in renewalRenewal considers stability or slowed decline on ALSFRS-R (no rapid progression on therapy) and excludes patients with cumulative ALSFRS-R ≤3

Clinical contextAlthough the Phase 3 trial did not meet the primary ALSFRS-R endpoint, ALSFRS-R remains a key functional outcome measure referenced in coverage and renewal decisions

inv-15: Plasma neurofilament light chain (NfL) — biomarker definition and role for baseline and response measurement

DefinitionPlasma neurofilament light chain (NfL) is a biomarker of axonal injury and neurodegeneration used to assess treatment response in SOD1-ALS

Role for baseline measurementBaseline plasma NfL must be documented prior to therapy initiation as part of eligibility requirements

Role for renewal/responseImprovement (reduction) in plasma NfL compared to baseline is required for renewal and is considered evidence of biomarker response

Regulatory contextFDA approval for Qalsody was based on a reduction in plasma NfL, which the FDA considers reasonably likely to predict clinical benefit

OpenPayer

Qalsody (tofersen) (Intrathecal) — Coverage Criteria

Coverage criteria for Qalsody (tofersen)

Billing and diagnosis codes

What providers must do

Background on Qalsody (tofersen)

Definitions and clinical measures