We develop Medical Policies to provide guidance to Members and Providers. This Medical Policy relates only to the services or supplies described in it. The existence of a Medical Policy is not an authorization, certification, explanation of benefits or a contract for the service (or supply) that is referenced in the Medical Policy. For a determination of the benefits that a Member is entitled to receive under his or her health plan, the Member's health plan must be reviewed. If there is a conflict between the Medical Policy and a health plan or government program (e.g., TennCare), the express terms of the health plan or government program will govern.

POLICY

INDICATIONS

The indications below including FDA-approved indications and compendial uses are considered a covered benefit provided that all the approval criteria are met and the member has no exclusions to the prescribed therapy.

FDA-Approved Indications

Lyfgenia is indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events.

Limitations of Use:

Following treatment with Lyfgenia, patients with α-thalassemia trait (-α3.7/-α3.7) may experience anemia with erythroid dysplasia that may require chronic red blood cell transfusions. Lyfgenia has not been studied in patients with more than two α-globin gene deletions.

All other indications are considered experimental/investigational and not medically necessary.

DOCUMENTATION

Submission of the following information is necessary to initiate the prior authorization review:

Molecular or genetic testing results documenting sickle cell disease genotype
Chart notes or medical records documenting history of severe vaso-occlusive episodes

PRESCRIBER SPECIALTIES

This medication must be prescribed by or in consultation with a hematologist.

COVERAGE CRITERIA

Sickle Cell Disease

Authorization of one dose total may be granted for sickle cell disease when all of the following criteria are met:

Member is 12 years of age or older.

Member has a diagnosis of sickle cell disease with one of the following genotypes confirmed by molecular or genetic testing:
- β^s/β^s
- β^s/β⁰
- β^s/β⁺
Member has a documented history of at least 2 severe vaso-occlusive episodes per year during the previous two years (see Appendix for examples).
Member is eligible for a hematopoietic stem cell transplant (HSCT) but is unable to find a human leukocyte antigen (HLA)-matched related donor.
Member has not received a prior hematopoietic stem cell transplant (HSCT).
Member has not received Lyfgenia or any other gene therapy previously.
Member does not have more than two α-globin gene deletions.
Member meets one of the following:

Has experienced, at any time in the past, an inadequate response or intolerance to a trial of hydroxyurea
Has a contraindication to hydroxyurea

APPENDIX

Examples of Severe Vaso-Occlusive Events

Acute pain event requiring a visit to a medical facility and administration of pain medications (opioids or intravenous [IV] non-steroidal anti-inflammatory drugs [NSAIDs]) or RBC transfusions
Acute chest syndrome
Priapism lasting > 2 hours and requiring a visit to a medical facility
Splenic sequestration
Hepatic sequestration

APPLICABLE TENNESSEE STATE MANDATE REQUIREMENTS

BlueCross BlueShield of Tennessee’s Medical Policy complies with Tennessee Code Annotated Section 56-7-2352 regarding coverage of off-label indications of Food and Drug Administration (FDA) approved drugs when the off-label use is recognized in one of the statutorily recognized standard reference compendia or in the published peer-reviewed medical literature.

ADDITIONAL INFORMATION

For appropriate chemotherapy regimens, dosage information, contraindications, precautions, warnings, and monitoring information, please refer to one of the standard reference compendia (e.g., the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) published by the National Comprehensive Cancer Network®, Drugdex Evaluations of Micromedex Solutions at Truven Health, or The American Hospital Formulary Service Drug Information).

REFERENCES

Lyfgenia [package insert]. Somerville, MA: bluebird bio, Inc.; December 2023.
Walters JK, Krishnamurti L, Mapara MY, et al. Biologic and clinical efficacy of LentiGlobin for sickle cell disease. NEJM. 2022;386(7):617-628.
Evidence-Based Management of Sickle Cell Disease: Expert Panel Report, 2014. National Institutes of Health. Available at https://www.nhlbi.nih.gov/sites/default/files/media/docs/sickle-cell-disease-report%20020816_0.pdf. Accessed July 16, 2024.

ORIGINAL EFFECTIVE DATE:4/2/2024

MOST RECENT REVIEW DATE: 4/2/2025

ID_CHS

Policies included in the Medical Policy Manual are not intended to certify coverage availability. They are medical determinations about a particular technology, service, drug, etc. While a policy or technology may be medically necessary, it could be excluded in a member's benefit plan. Please check with the appropriate claims department to determine if the service in question is a covered service under a particular benefit plan. Use of the Medical Policy Manual is not intended to replace independent medical judgment for treatment of individuals. The content on this Web site is not intended to be a substitute for professional medical advice in any way. Always seek the advice of your physician or other qualified health care provider if you have questions regarding a medical condition or treatment.

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