Pharmacy Prior Authorization Criteria

Zynteglo (betibeglogene autotemcel) - PA, NF

Indications for Prior Authorization

Zynteglo (betibeglogene autotemcel)

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

Criteria

Zynteglo

Prior Authorization

Length of Approval: 1 Time Authorization in Lifetime

• 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)
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
• Patient is 4 years of age or older [A]
AND
• Provider attests that patient is clinically stable and eligible to undergo hematopoietic stem cell transplant (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 [1-4]:
• 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
• Prescribed by a stem cell transplant specialist
AND
• Patient has never received Zynteglo treatment in their lifetime

Zynteglo

Non Formulary

Length of Approval: 1 Time Authorization in Lifetime

• 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)
AND
• Submission of medical records (e.g., chart notes) documenting 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
• Patient is 4 years of age or older [A]
AND
• Provider attests that patient is clinically stable and eligible to undergo hematopoietic stem cell transplant (HSCT)
AND
• Submission of medical records (e.g., chart notes) documenting 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
• Submission of medical records (e.g., chart notes) documenting patient does not have any of the following [1-4]:
• 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
• Prescribed by a stem cell transplant specialist
AND
• Patient has never received Zynteglo treatment in their lifetime

P & T Revisions

2024-10-24, 2023-10-19, 2022-11-29, 2022-11-17, 2022-10-07

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
5. Per clinical consult with pediatric hematologist/oncologist. October 7, 2022.

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End Notes

1. The safety and efficacy of Zynteglo in children less than 4 years of age have not been established. [1]

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Revision History

• 2024-10-24: 2024 Annual Review. No updates
• 2023-10-19: Annual review - no criteria changes.
• 2022-11-29: update guideline
• 2022-11-17: Update to remove requirement that patient is ineligible for allogeneic HSCT with a sibling donor and that patient has not received prior gene therapy/HSCT per P&T committee feedback.
• 2022-10-07: New PA program for Zynteglo.

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