CurrentViva HealthPolicy IC-0704

Qalsody (tofersen) intrathecal therapy for SOD1-mutant amyotrophic lateral sclerosis (ALS)

Clinical coverage and prior authorization criteria for intrathecal Qalsody (tofersen) in adult patients with SOD1-mutant amyotrophic lateral sclerosis (ALS); includes initial and renewal requirements, dosing, billing codes, and allowable units.

Policy Summary

PayerViva Health

PolicyQalsody (tofersen) intrathecal therapy for SOD1-mutant amyotrophic lateral sclerosis (ALS)

Policy CodePolicy IC-0704

Change TypeNo material changes

Effective Date06/01/2023

Next Review Date

Key ActionObtain prior authorization and document baseline SOD1 mutation status, plasma NfL, %SVC, and item-level ALSFRS-R; renewals require demonstrated NfL improvement, clinical stability or slowed decline, and absence of unacceptable toxicity.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

18 monthsInitial + first renewal authorization window (6 month initial then 12 month renewal)

100 mgDose per billing unit described (100 billable units = 100 mg)

>= 65%Minimum slow vital capacity (%SVC) required

ALSFRS-R ≥ 24Minimum implied functional baseline (score of 2 or better on each item)

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Initial Approval Criteria

Coverage is provided when ALL of the following are met:

ALL of the following

Patient is at least 18 years of age
Patient has a baseline measure of the plasma neurofilament light chain (NfL)
ALS-specific requirements (ALL):
- Diagnosis of clinically definite or probable ALS based on El Escorial revised criteria, Gold Coast criteria, or Awaji criteria
- Presence of a mutation in the superoxide dismutase 1 (SOD1) gene
- Slow vital capacity (%SVC) ≥ 65%>= 65%
- Baseline documentation of retained functionality for most activities of daily living: score of 2 or better on each individual item of the ALSFRS-R>= 2 on each item
  This corresponds to a minimum possible cumulative ALSFRS-R of 24 if every item is >=2

Renewal Criteria

Coverage renewal may be approved when ALL of the following are met:

ALL of the following

Patient continues to meet the indication-specific criteria from Initial Approval (section III)
Absence of unacceptable toxicity from the drug (examples: serious myelitis and radiculitis, papilledema and elevated intracranial pressure, aseptic meningitis, etc.)
Unacceptable toxicity may lead to non-renewal
Patient has improvement in plasma neurofilament light chain (NfL) levels compared to baseline
Requires documented comparison to baseline NfL
Patient has responded to therapy compared to pretreatment baseline with disease stability or mild progression indicating a slowing of decline on the ALSFRS-R (no rapid disease progression while on therapy)

Prior Authorization, Documentation, and Denial Risks

Prior Authorization

Prior authorization required

Prior authorization must be obtained. Initial approval is granted for 6 months; renewals may be granted every 12 months thereafter.

Affected billing code: J1304

Documentation Required

Required baseline and follow-up documentation

Document baseline and follow-up measures to support coverage decisions.

Baseline: plasma NfL level
Baseline: SOD1 mutation status
Baseline: diagnostic criteria used (El Escorial, Gold Coast, or Awaji)
Baseline: %SVC (slow vital capacity) ≥ 65%
Baseline: ALSFRS-R item-level scores (score of 2 or better on each individual item)
Renewal: documented improvement in plasma NfL compared to baseline
Renewal: ALSFRS-R course showing stability or slowed decline (no rapid disease progression on therapy)
Renewal: documentation of absence of unacceptable toxicity

Denial Risk

Renewal denial conditions

Renewal may be denied for specified safety, biomarker, or clinical progression reasons.

Unacceptable toxicity (examples: serious myelitis and radiculitis, papilledema with elevated intracranial pressure, aseptic meningitis)
Lack of improvement in plasma NfL compared to baseline
Rapid disease progression while on therapy (failure to demonstrate stability or slowed decline on ALSFRS-R)
Cumulative ALSFRS-R score of ≤ 3

Clinical Background

Qalsody (tofersen) is an antisense oligonucleotide approved for the treatment of amyotrophic lateral sclerosis (ALS) associated with a pathogenic variant in the SOD1 gene and is administered via intrathecal injection. The policy requires patients to be adults (age ≥ 18 years) and to meet established diagnostic standards for ALS, specifically diagnosis of clinically definite or probable ALS based on the El Escorial revised criteria, Gold Coast criteria, or Awaji criteria.

Baseline assessment must include a measure of the plasma neurofilament light chain (NfL) and documentation of respiratory function as percent predicted slow vital capacity (%SVC), with a required baseline of ≥ 65% SVC prior to initiation. Functional status at baseline must be documented using the ALS Functional Rating Scale – Revised (ALSFRS‑R) with item-level scores showing retained function (a score of 2 or better on each individual item, which corresponds to an implied minimum cumulative score of 24).

During treatment, follow-up monitoring of plasma NfL is required and renewal of coverage depends on documented biochemical response (improvement in NfL compared with baseline), clinical response on the ALSFRS‑R showing stability or slowed decline (no rapid progression while on therapy), and absence of unacceptable toxicity such as serious myelitis/radiculitis, papilledema with elevated intracranial pressure, or aseptic meningitis. A cumulative ALSFRS‑R score of ≤ 3 is exclusionary for continued coverage.

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Defined Terms

Definitions

ALSFRS-RAmyotrophic Lateral Sclerosis Functional Rating Scale - Revised; 12-item questionnaire each scored 0-4, cumulative maximum 48. Used to assess function across bulbar, fine motor, gross motor, and respiratory domains.

NfLPlasma neurofilament light chain, a biomarker measured at baseline and during therapy to assess biochemical response.

%SVCPercent predicted slow vital capacity, a measure of respiratory function; policy requires >= 65% for initiation.

Unacceptable toxicitySerious adverse events such as myelitis, radiculitis, papilledema with elevated intracranial pressure, aseptic meningitis, etc., that would preclude continuation.