ZYNTEGLO (betibeglogene autotemcel)

ZYNTEGLO (betibeglogene autotemcel)

Office-Administration – intravenous (IV) infusion

Diagnosis considered for coverage:

Beta-thalassemia: Indicated for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell (RBC) transfusions.

Coverage Criteria:

Diagnosis of transfusion-dependent beta-thalassemia as confirmed by the presence of a mutation at both alleles of the β-globin gene (i.e., β0/β0, β0/β+, β+/β+, β0/βE):

• Patient is 4 years of age or older; AND
• Prescribed by a stem cell transplant specialist; AND
• One of the following:
  • Patient has a history of transfusions of at least 100 mL/kg/year of packed red blood cells (pRBCs)
  • Patient requires 8 or more red blood cell (RBC) transfusions per year; AND
• Provider attests that patient is clinically stable and eligible to undergo hematopoietic stem cell transplant (HSCT) and has not received any prior gene therapy or HSCT; AND 
• Patient has obtained a negative test result for all of the following prior to cell collection:
  • Hepatitis B virus (HBV) 
  • Hepatitis C virus (HCV) 
  • Human T-lymphotrophic virus 1 & 2 (HTLV-1/HTLV-2) 
  • Human immunodeficiency virus (HIV); AND
• Patient is able to provide an adequate number of cells to meet the minimum recommended dose of 5 x 10^6 CD34+ cells/kg; AND
• Patient does not have any of the following:
  • Severely elevated iron in the heart (e.g., patients with cardiac T2* less than 10 msec by MRI) 
  • Advanced liver disease 
  • MRI results of the liver demonstrating liver iron content greater than or equal to 15 mg/g (unless biopsy confirms absence of advanced disease); AND
• BOTH of the following:
  • Iron chelation therapy (e.g., deferoxamine, deferasirox) will be discontinued for at least 7 days prior to initiating myeloablative conditioning therapy 
  • Prophylactic HIV anti-retroviral medications (e.g., Truvada, Descovy) or hydroxyurea will be discontinued for at least one month prior to mobilization (or for the expected duration for elimination of those medications); AND
• Patient has never received Zynteglo treatment in their lifetime

Coverage Duration: 

• One Time Authorization in Lifetime.

Authorization is not covered for the following:

The use of this product 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.

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Additional Information: 

• The target number of CD34+ cells to be collected is ≥ 12 × 106 CD34+ cells/kg.

Policy Updates:

• 02/15/2023 – New utilization management policy approved by WHA P&T.

References:

1. Zynteglo Prescribing Information. Bluebird Bio, Inc. Somerville, MA. August 2022. 
2. Galanello R and Origa R. Beta-thalassemia. Orphanet J Rare Dis. 2010 May 21;5:11. Available at: https://ojrd.biomedcentral.com/articles/10.1186/1750-1172-5-11. Accessed October 2022. 
3. Locatelli F, Thompson AA, Kwiatkowski JL, et al. Betibeglogene Autotemcel Gene Therapy for Non-β(0)/β(0) Genotype β-Thalassemia. N Engl J Med. 2022 Feb 3;386(5):415-427. doi: 10.1056/NEJMoa2113206. Epub 2021 Dec 11. 
4. Beaudoin FL, Richardson M, Synnott PG, et al. Betibeglogene Autotemcel for Beta Thalassemia: Effectiveness and Value; Final Evidence Report. Institute for Clinical and Economic Review, July 19, 2022. https://icer.org/beta-thalassemia-2022/#timeline