QALSODY (tofersen)

QALSODY (tofersen)

Office Administration – intrathecally using lumbar puncture

Diagnosis considered for coverage:

• Indicated for treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. This indication is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with Qalsody. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).

Coverage Criteria:

For diagnosis of ALS with SOD1 gene in adults:

• Dose does not exceed the recommended dose of 100mg (15mL) per administration, AND
• Molecular genetic testing that confirms mutation in the SOD1 gene, AND
• Patient is 18 years old or older, AND
• Prescribed by or in consultation with a neurologist with expertise in the diagnosis of ALS, AND
• Patient’s baseline functional ability has been documented prior to initiating treatment (e.g., speech, walking, climbing stairs, etc.), AND
• Patient has a percent (%) slow vital capacity (%SVC) greater than or equal to 50% at the start of treatment, AND
• Patient does not require permanent noninvasive ventilation or invasive ventilation 

Reauthorization Criteria:

For diagnosis of ALS with SOD1 gene:

• Dose does not exceed the recommended dose of 100mg (15mL) per administration, AND
• Documentation of slowed disease progression from baseline

Coverage Duration: 

Initial: 6 months
Reauthorization: 6 months

Dosing:

• Office administered injection 100mg/15mL (6.7 mg/mL) solution in a single-dose vial per administration
• 3 loading doses- Every 14 days
• Administer maintenance dose 28 days thereafter
• After 10 mL of CSF removed, tofersen is administered as intrathecal bolus over 1 to 3 mins. 
• Administered immediately after withdrawn into syringe (within 4 hours of removal from vial)

Authorization is not covered for the following:

The use of this drug for indications not listed in this policy does not meet the coverage criteria established by the Western Health Advantage (WHA) Pharmacy and Therapeutics (P&T) Committee.

Additional Information: 

• Approximately 2% of ALS cases are associated with mutations in the gene encoding SOD1
• Tofersen was granted orphan drug, priority review, and fast track designations (FDA news release 2023).
• The American Academy of Neurology (AAN) practice guidelines issued in 2009 (reaffirmed January 11, 2020) recommend riluzole to slow progression and for patients who develop fatigue while taking riluzole, withholding the drug may be considered.

Policy Updates:

• 12/1/2023 (policy effective date) – New policy approved by WHA P&T Committee. (P&T, 11/14/2023) (P&T meeting date) 

References:

1. Qalsody Prescribing Information. Biogen MA Inc. Cambridge, MA. April 2023.
2. Miller TM, Cudkowicz ME, Genge A, et al. Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. New England Journal of Medicine. 2022;387(12):1099-1110. 
3. Qalsody. IBM Micromedex Solutions. Truven Health Analytics, Inc. Ann Arbor, MI. Accessed September 5, 2023. http://www.micromedexsolutions.com.