CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Casgevy (exagamglogene autotemcel) intravenous gene therapy

Defines coverage, medical necessity criteria, quantity/dosing limits, authorization length, and billing codes for Casgevy for Medicaid, Commercial, and Medicare‑Medicaid Plan members (age 12-35) per Neighborhood Health Plan of Rhode Island.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyCasgevy (exagamglogene autotemcel) intravenous gene therapy

Policy CodePolicy N/A

Change TypeClarified (administrative review dates)

Effective Date05/01/2024

Next Review Date

Key ActionSubmission of medical records related to medical necessity is REQUIRED on all requests; provide documentation via PA web portal or fax.

POLICY UPDATE CHANGES

Policy effective 05/01/2024 with review dates listed 03/20/2024, 10/09/2024, 04/08/2025.

1Authorized treatment course per member

>=3.0E6Minimum CD34+ cells/kg

12-35Covered age range

J3392, NDCBilling codes provided

Coverage Summary & Scope

General Initial Approval Criteria

Covered when ALL of the following are met:

ALL of the following

Patient is at least 12-35 years of age.
Provider has considered use of prophylaxis therapy for seizures prior to initiating myeloablative conditioning.
Patient has been screened and found negative for HBV, HCV, and HIV-1/HIV-2 prior to collection of cells (leukapheresis).
Must not be administered concurrently with live vaccines while immunosuppressed.
Patient does not have a history of hypersensitivity to dimethyl sulfoxide (DMSO) or dextran 40.
Patient has not received other gene therapies (e.g., lovotibeglogene autotemcel, betibeglogene autotemcel).
Patient will not receive therapy concomitantly with any of the following:
- Iron chelators for 7 days prior to mobilization and 6 months post-treatment (3 months post-treatment for non‑myelosuppressive iron chelators).
- Disease‑modifying agents (e.g., hydroxyurea or crizanlizumab) for at least 8 weeks prior to mobilization and conditioning.
Patient does not have a known 10/10 HLA matched related donor willing to participate in an allogeneic HSCT.
Patient is a candidate for autologous hematopoietic stem cell transplant (no prohibitive bone marrow, lung, heart, or liver dysfunction).
Patient does not have a history of previous hematopoietic stem cell transplant (HSCT).
Patient does not have a potential contraindication to any required product or procedure, including but not limited to:
- Plerixafor.
- Busulfan.
- Red blood cell infusion.
Patient will undergo treatment at a manufacturer‑approved Authorized Treatment Center (ATC).
Casgevy is prescribed by, or in consultation with, a hematology specialist.

Sickle Cell Disease Indication Criteria

Patient has a confirmed diagnosis of sickle cell disease (including genotypes βS/βS, βS/β0, or βS/β+), by hemoglobin assay showing significant HbS with or without an additional abnormal β‑globin chain variant, OR by molecular genetic testing showing biallelic HBB pathogenic variants where at least one allele is p.Glu6Val (sickle mutation).
Patient has symptomatic disease despite treatment with hydroxyurea and formulary add‑on therapy (e.g., crizanlizumab) where clinically appropriate.
Patient experienced two or more vaso‑occlusive events/crises (VOE/VOC) per year in the previous two years while adherent to the above therapy. (VOE/VOC defined as event requiring medical facility visit resulting in diagnosis documented for acute pain, acute chest syndrome, acute splenic or hepatic sequestration, priapism >2 hours, and necessitating interventions such as opioid pain management, NSAIDs, or RBC transfusion.)
Patient will be transfused prior to apheresis to a total hemoglobin ≤11 g/dL and HbS level <30%, and transfusion will occur at least 8 weeks prior to initiation of myeloablative conditioning.

Beta Thalassemia Indication Criteria

Patient has a documented diagnosis of homozygous beta thalassemia or compound heterozygous beta thalassemia (including β‑thalassemia/hemoglobin E) confirmed by either: HBB sequence analysis showing biallelic pathogenic variants; OR clinical/laboratory findings (severe microcytic hypochromic anemia without iron deficiency, anisopoikilocytosis with nucleated RBCs, hemoglobin analysis showing absent or markedly reduced HbA with increased HbA2 ± increased HbF).
Patient has transfusion‑dependent disease defined as history of transfusions ≥100 mL/kg/year or ≥10 units/year of packed red blood cells in the 2 years preceding therapy.
Patient will be transfused prior to apheresis to a total hemoglobin ≥11 g/dL for 60 days prior to myeloablative conditioning.
Patient must NOT have any of the following severe comorbidities:
- Cardiac iron overload (cardiac T2* < 10 msec by MRI) or left ventricular ejection fraction <45% by echocardiogram.

Exclusions / Non-coverage

The following are exclusions / non‑coverage criteria:

ANY of the following

Patients outside the age range of 12–35 years.
Patients who have previously received other gene therapies (e.g., lovotibeglogene autotemcel, betibeglogene autotemcel).
Patients with a known 10/10 HLA matched related donor willing to proceed with allogeneic HSCT (i.e., alternative curative option available).
Patients with contraindications to required products or procedures for successful gene therapy (see General Initial Approval Criteria).
Patients with severely elevated cardiac iron (cardiac T2* <10 msec) or LVEF <45%, or advanced liver disease as defined in Beta Thalassemia criteria.

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Prior Authorization, Documentation & Billing Requirements

Prior Authorization

Submit medical records and prior authorization

Submission of medical records (chart notes) related to medical necessity is REQUIRED on all requests. Provide documentation related to diagnosis, step therapy, and clinical markers (e.g., genetic/mutational testing) supporting initiation. Submit documents via direct upload through the PA web portal or by fax.

Chart notes documenting diagnosis and clinical criteria
Genetic / mutational testing results (when applicable)
Documentation of prior therapies and step therapy adherence

Documentation Required

Infection screening prior to leukapheresis

Document negative screening results for hepatitis B virus (HBV), hepatitis C virus (HCV), and human immunodeficiency virus (HIV-1/HIV-2) prior to collection of cells (leukapheresis) in accordance with clinical guidelines.

Documentation Required

Transfusion and lab goals documentation

Document transfusion and laboratory targets prior to apheresis per indication: for sickle cell disease, transfuse prior to apheresis to achieve total hemoglobin ≤ 11 g/dL and HbS < 30%, and transfuse at least 8 weeks prior to initiation of myeloablative conditioning; for transfusion-dependent beta thalassemia, transfuse prior to apheresis to maintain total hemoglobin ≥ 11 g/dL for 60 days prior to myeloablative conditioning.

SCD pre-apheresis goals: Hb ≤ 11 g/dL and HbS < 30%; transfused ≥ 8 weeks before conditioning
Beta thalassemia pre-apheresis goal: Hb ≥ 11 g/dL for 60 days prior to conditioning

Documentation Required

Treatment at Authorized Treatment Center

Document that the patient will undergo treatment at a manufacturer‑approved Authorized Treatment Center (ATC).

Documentation Required

Specialist involvement

Document that Casgevy is prescribed by, or in consultation with, a hematology specialist (prescribing clinician specialty or consultation note).

Billing Rule

Single dose authorization / quantity limit

Coverage is provided for one treatment course (single dose) only. Quantity limit requires a single dose containing a minimum of 3.0 × 10^6 CD34+ cells/kg in multiple vials.

HCPCS: J3392 - Injection, exagamglogene autotemcel, 1 mg; 1 billable unit = 1 treatment
NDC: 51167-0290-xx (Casgevy containing a minimum of 3.0 × 10^6 CD34+ cells/kg)

Applicable Codes

HCPCS (billing)HCPCSCovered

J3392

Injection, exagamglogene autotemcel, 1 mg; 1 billable unit = 1 treatment

NDC (product)NDCCovered

51167-0290-xx

Casgevy containing a minimum of 3.0 × 10^6 CD34+ cells/kg of body weight, in multiple vials supplied in vial(s) packaged in carton(s)

Key thresholds and limits include a required minimum CD34+ cell dose of >= 3.0 × 10^6 CD34+ cells/kg per single dose (product supplied in multiple vials), SCD pre‑apheresis transfusion goals of total Hb ≤ 11 g/dL and HbS <30%, and beta thalassemia pre‑apheresis transfusion requirement of total Hb ≥ 11 g/dL for 60 days prior to myeloablative conditioning. For transfusion‑dependent beta thalassemia, documented transfusion history must meet >=100 mL/kg/year or ≥10 units/year pRBCs in the 2 years preceding therapy. Coverage is limited to a single treatment course (one dose) of Casgevy and may not be renewed.

Therapy & Definitions

Casgevy is a CRISPR‑based, autologous gene‑edited hematopoietic stem cell (HSC) therapy (exagamglogene autotemcel) designed to treat underlying β‑hemoglobinopathies by gene editing of a patient’s own CD34+ HSCs. It is indicated for treatment of sickle cell disease and transfusion‑dependent beta thalassemia and is provided as a single intravenous dose containing a patient‑specific suspension of CD34+ cells to achieve the recommended minimum cell dose.

Term	Definition
VOE/VOC
An event requiring a visit to a medical facility resulting in diagnosis documented due to acute pain, acute chest syndrome, acute splenic sequestration, acute hepatic sequestration, priapism >2 hours and necessitating interventions (opioids, NSAIDs, RBC transfusion, etc.).
Authorized Treatment Center (ATC)
Manufacturer approved center where Casgevy treatment must be performed.

OpenPayer

Casgevy (exagamglogene autotemcel) intravenous gene therapy

Coverage Summary & Scope

Prior Authorization, Documentation & Billing Requirements

Applicable Codes

Efficacy, Safety & Trial Outcomes

Therapy & Definitions

Policy Changes & Review Dates