Last Review Date: 07/02/2024

Date of Origin: 06/01/2023

Dates Reviewed: 06/2023, 07/2024

1. Length of Authorization

Coverage will be provided for 6 months for initial approval and may be renewed every 12 months thereafter.

2. Dosing Limits

1. Quantity Limit (max daily dose) [NDC Unit]:

• Qalsody 100 mg/15 mL single-dose vial: 1 vial every 14 days for 3 doses, then 1 vial every 28 days thereafter

2. Max Units (per dose and over time) [HCPCS Unit]:

• Initial dose: 100 billable units (100 mg) every 14 days, for 3 doses only
• Subsequent doses: 100 billable units (100 mg) every 28 days thereafter

3. Initial Approval Criteria ¹

Coverage is provided in the following conditions:

• Patient is at least 18 years of age; AND
• Patient has a baseline measure of the plasma neurofilament light chain (NfL); AND

Amyotrophic Lateral Sclerosis (ALS) † Ф ^1,2,7,8

• Patient has a diagnosis of clinically definite or probable ALS based on El Escorial revised criteria or Awaji criteria; AND
• Patient has the presence of a mutation in the superoxide dismutase 1 (SOD1) gene: 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 or better on each* individual item of the ALS Functional Rating Scale – Revised (ALSFRS-R)]

*Note: the ALSFRS-R is a 12-item questionnaire assessing functional disease progression across four domains including bulbar, fine motor, gross motor, and respiratory. Each item is scored on a five-point ordinal scale from 0 (loss or significant impairment) up to 4 (normal function) with a possible cumulative score of 48. A score of 2 or better on each item would correspond to a minimum ALSFRS-R score of 24.

† FDA Approved Indication(s); ‡ Compendia Recommended Indication(s); Ф Orphan Drug

4. Renewal Criteria ^1,3

Coverage can be renewed based upon the following criteria:

• Patient continues to meet the indication-specific relevant criteria identified in section III; AND
• Absence of unacceptable toxicity from the drug. Examples of unacceptable toxicity include: serious myelitis and radiculitis, papilledema and elevated cranial pressure, aseptic meningitis, etc.; AND
• Patient has improvement in the plasma neurofilament light chain (NfL) levels 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 score* on the ALSFRS-R of ≤ 3 (The cumulative possible score on the ALSFRS-R is 48; a cumulative score of 3 indicates loss/significant impairment [i.e., item score of zero] in nine or more items on the 12-item questionnaire)

*Note: the ALSFRS-R is a 12-item questionnaire assessing functional disease progression across four domains including bulbar, fine motor, gross motor, and respiratory. Each item is scored on a five-point ordinal scale from 0 (loss or significant impairment) up to 4 (normal function) with a possible cumulative score of 48. A score of 2 or better on each item would correspond to a minimum ALSFRS-R score of 24.

5. Dosage/Administration ¹

6. Billing Code/Availability Information

HCPCS Code:

• J1304 – Injection, tofersen, 1 mg; 1 billable unit = 1 mg

NDC:

• 100 mg/15 mL solution in a single-dose glass vial (preservative free): 64406-0109-xx

7. References

1. Qalsody [package insert]. Cambridge, MA; Biogen MA, Inc; April 2023. Accessed May 2024.
2. Miller TM, Cudkowicz ME, Genge A, et al; VALOR and OLE Working Group. Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. N Engl J Med. 2022 Sep 22;387(12):1099-1110. doi: 10.1056/NEJMoa2204705.
3. Cedarbaum JM, Stambler N, Malta E, et al. The ALSFRS-R: a revised ALS functional rating scale that incorporates assessments of respiratory function. BDNF ALS Study Group (Phase III). J Neurol Sci. 1999 Oct 31;169(1-2):13-21.
4. Miller RG, Jackson CE, Kasarskis EJ, et al. Practice parameter update: the care of the patient with amyotrophic lateral sclerosis: drug, nutritional, and respiratory therapies (an evidence-based review): report of the Quality Standards Subcommittee of the American Academy of Neurology. Neurology. 2009 Oct 13;73(15):1218-26.
5. Siddique N, Siddique T. Amyotrophic Lateral Sclerosis Overview. GeneReviews. Initial Posting: March 23, 2021; Last Revision: September 28, 2023; Accessed on May 28, 2024. http://www.ncbi.nlm.nih.gov/books/NBK1450/.
6. Hardiman O, van den Berg LH, Kiernan MC. Clinical diagnosis and management of amyotrophic lateral sclerosis. Nat Rev Neurol. 2011 Oct 11;7(11):639-49.
7. Costa J, Swash M, de Carvalho M. Awaji criteria for the diagnosis of amyotrophic lateral sclerosis: a systematic review. Arch Neurol. 2012 Nov;69(11):1410-6.
8. Pinto S, de Carvalho M. SVC Is a Marker of Respiratory Decline Function, Similar to FVC, in Patients With ALS. Front Neurol. 2019 Feb 28;10:109. doi: 10.3389/fneur.2019.00109. PMID: 30873101; PMCID: PMC6403463.

Appendix 1 – Covered Diagnosis Codes

Appendix 2 – Centers for Medicare and Medicaid Services (CMS)

The preceding information is intended for non-Medicare coverage determinations. Medicare coverage for outpatient (Part B) drugs is outlined in the Medicare Benefit Policy Manual (Pub. 100-2), Chapter 15, §50 Drugs and Biologicals. In addition, National Coverage Determinations (NCDs) and/or Local Coverage Determinations (LCDs) may exist and compliance with these policies is required where applicable. Local Coverage Articles (LCAs) may also exist for claims payment purposes or to clarify benefit eligibility under Part B for drugs which may be self-administered. The following link may be used to search for NCD, LCD, or LCA documents: https://www.cms.gov/medicare-coverage-database/search.aspx. Additional indications, including any preceding information, may be applied at the discretion of the health plan.

Medicare Part B Covered Diagnosis Codes (applicable to existing NCD/LCD/LCA): N/A