LENMELDY (atidarsagene autotemcel)

LENMELDY (atidarsagene autotemcel)

Office-Administration – intravenous (IV) infusion

Diagnosis considered for coverage:

Metachromatic Leukodystrophy (MLD): Indicated for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD).

Coverage Criteria:

For diagnosis of MLD:

• Diagnosis of metachromatic leukodystrophy (MLD); AND
• Molecular genetic testing confirms mutation in the arylsulfatase A (ARSA) gene; AND 
• Disease is one of the following: 
  • Pre-symptomatic late infantile (PSLI) as confirmed by both of the following: 
    • Disease onset at less than or equal to 30 months of age, and 
    • One of the following: 
      • Absence of neurological signs and symptoms of MLD (e.g., peripheral neuropathy, gait difficulties, hypotonia) 
      • Abnormal reflexes or abnormalities on brain magnetic resonance imaging (MRI) and/or nerve conduction tests not associated with functional impairment (e.g., no tremor, no peripheral ataxia) OR
  • Pre-symptomatic early juvenile (PSEJ) as confirmed by both of the following: 
    • Disease onset at greater than 30 months and less than 7 years of age, and 
    • One of the following: 
      • Absence of neurological signs and symptoms of MLD (e.g., peripheral neuropathy, gait difficulties, hypotonia) 
      • Abnormal reflexes or abnormalities on brain magnetic resonance imaging (MRI) and/or nerve conduction tests not associated with functional impairment (e.g., no tremor, no peripheral ataxia) OR
  • Early-symptomatic early juvenile (ESEJ) as confirmed by all of the following: 
    • Disease onset at greater than 30 months and less than 7 years of age 
    • Gross motor function classification (GMFC)-MLD score less than or equal to 1 
    • Intelligence quotient (IQ) of greater than or equal to 85; AND
• Prescribed by a specialist with expertise in MLD at an authorized treatment center; AND
• Both of the following: 
  • Patient has never received Lenmeldy treatment in their lifetime 
  • Patient has never received prior hematopoietic stem cell transplant (HSCT)

Coverage Duration:

• 1 time authorization in lifetime

Dosing:

For diagnosis of MLD:

• Administration consists of HSC mobilization & apheresis, manufacturing (transduction), myeloablative conditioning, infusion, and post-administration (engraftment)
• Dose is based on MLD subtype and child’s weight at the time of infusion: 
  • PSLI (4.2 to 30 X 106 CD34+ cells/kg)
  • PSEJ (9.0 to 30 X 106 CD34+ cells/kg)
  • ESEJ (6.6 to 30 X 106 CD34+ cells/kg)

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: 

• MLD is a rare inherited lysosomal storage disease due to a deficiency in the arylsulfatase A [ARSA] enzyme resulting in the accumulation of sulfatides, leading to neurodegeneration.
• Lenmeldy is the first FDA-approved therapy for the treatment of PSLI, PSEJ, and ESEJ MLD. 
• Lenmeldy is a gene therapy utilizing autologous HSPCs enriched with CD34+ cells, then transduced ex vivo with an HIV-1-based LVV that has been modified to carry the ARSA cDNA sequence, driving ARSA enzyme production, leading to breakdown of sulfatides.
• Lenmeldy is administered as a one-time IV infusion to add functional copies of the human ARSA gene into the patient’s HSPCs.
• The FDA has implemented post-marketing requirements to evaluate long-term efficacy and safety related to gene therapies.

Policy Updates:

• 09/01/2024 – New policy for Lenmeldy approved by WHA P&T Committee. (P&T, 08/20/2024)

References:

1. Lenmeldy Prescribing Information. Orchard Therapeutics. Boston, MA. March 2024.