CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Mylotarg (gemtuzumab ozogamicin) — Coverage Criteria

Governance of authorization and medical necessity criteria for Mylotarg (gemtuzumab ozogamicin) for treatment of cancer (primarily CD33-positive AML) for Neighborhood Health Plan of Rhode Island members; applies to providers submitting medication requests to the payer/UM vendor.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyMylotarg (gemtuzumab ozogamicin) — Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateAugust 30, 2024

Next Review DateN/A

Key ActionSubmit prior authorization to UM; approvals issued by the Utilization Management Committee and requests must be supported by FDA labeling, recognized compendia, or accepted peer-reviewed literature.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

2024-08-14Most recent committee approval date

14Committee review dates listed

1 mo+Allowed use in newly diagnosed AML (min age)

2 yrs+Allowed use in relapsed/refractory AML (min age)

3 mg/m2

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Coverage Criteria for Mylotarg (gemtuzumab ozogamicin)

Covered Indications - AML

Covered when ALL of the following are met

AML indication and prior receipt: Member has CD33-positive acute myeloid leukemia and Mylotarg (gemtuzumab ozogamicin) is being used as a single agent OR in combination with chemotherapy for newly diagnosed AML (age >= 1 month) OR relapsed/refractory AML (age >= 2 years) provided the patient has not previously received Mylotarg.age limits as stated

Applies only if Mylotarg has not been received previously in relapsed/refractory patients

Requests for Mylotarg (gemtuzumab ozogamicin) will be excluded when the member has experienced disease progression on or following Mylotarg or a Mylotarg-containing regimen. In addition, dosing that exceeds the policy’s single‑dose limits is not approvable: for adults, 3 mg/m2 (maximum 4.5 mg) when given in combination or 6 mg/m2 when given as a single agent; for pediatric members ≥0.6 m2 BSA, 3 mg/m2; and for pediatric members with BSA <0.6 m2, 0.1 mg/kg.

Use of Mylotarg in regimens that lack Level 1 evidence (randomized clinical trials and/or meta-analyses) is not supported by this policy and is considered not medically necessary. Examples specified in the policy include: induction regimens for patients <60 years that combine Fludarabine + HiDAC + idarubicin + G-CSF + gemtuzumab ozogamicin, and single‑agent Mylotarg for induction/consolidation in patients ≥60 years. These positions reflect the absence of high‑level evidence showing superior outcomes compared with Evolent‑recommended alternatives and pathways.

Requests for investigational or off‑label uses must be supported by sufficient evidence as defined in the policy (representation of the patient population and regimen in the published evidence, clinically meaningful outcomes per ASCO thresholds, and appropriate study design). Uses not meeting these evidence requirements or not supported by CMS‑recognized compendia or acceptable peer‑reviewed literature may be denied.

Regimen	Age / Setting	Policy coverage stance
Fludarabine + HiDAC + idarubicin + G-CSF + gemtuzumab ozogamicin
< 60 years — induction therapy
Induction regimen not supported by policy (lack of Level 1 evidence)
Single-agent gemtuzumab ozogamicin
>= 60 years — induction/consolidation therapy
Induction/consolidation regimen not supported by policy (lack of Level 1 evidence)

Permitted Lines of Therapy

first-line | relapsed/refractory

Line of therapy: Mylotarg may be used for newly diagnosed AML (age >= 1 month) and for relapsed/refractory AML (age >= 2 years) provided the patient has not previously received Mylotarg.

Biomarker and Disease Requirements

CD33 — requirement for CD33-positive disease

RequirementMember must have CD33-positive acute myeloid leukemia (AML).

Allowed settingsUse permitted as single agent or in combination with chemotherapy for newly diagnosed AML (age ≥1 month).

Relapsed/refractoryPermitted for relapsed/refractory AML in members age ≥2 years who have not previously received Mylotarg.

CD33-positive AML — definition and scope

DefinitionAcute myeloid leukemia expressing the CD33 antigen (CD33-positive AML).

Scope — age limitsNewly diagnosed AML: age 1 month and older; Relapsed/refractory AML: age 2 years and older (relapsed/refractory patients must be Mylotarg-naïve).

Dosing Limits and Coding Notes

Adult single dose limit (combination therapy)

Adult combination therapy single-dose limit3 mg/m2 per single dose (maximum single dose 4.5 mg).

ApplicabilityApplies when Mylotarg is given in combination with other chemotherapy agents to adult members.

Exclusion consequenceRequests exceeding this single-dose limit will be excluded per policy.

Adult single dose limit (single-agent)

Adult single-agent single-dose limit6 mg/m2 per single dose.

ApplicabilityApplies when Mylotarg is administered as a single agent to adult members.

Provider Requirements and Prior Authorization

Prior Authorization

Prior Authorization and Approval Authority

All requests for Mylotarg (gemtuzumab ozogamicin) require review by the Utilization Management (UM) department and final approval by the Utilization Management Committee. Evolent (acting for Neighborhood Health Plan of Rhode Island) is responsible for processing medication requests; medications not authorized by Evolent/UM may be denied and not reimbursed.

Final authorization authority: Utilization Management Committee
Initial clinical review performed by Utilization Management Department

Denial Risk

Disease progression on prior Mylotarg

Requests for Mylotarg will be excluded when the member has documented disease progression on or following prior treatment with Mylotarg or a Mylotarg-containing regimen.

Exclude requests following documented progression on prior Mylotarg or Mylotarg-containing therapy.

Regimens and Regimen-Specific Notes

Regimen details	Documented rationale / policy note	Coverage stance
Fludarabine + high-dose cytarabine (HiDAC) + idarubicin + granulocyte colony-stimulating factor (G-CSF) + gemtuzumab ozogamicin
Listed as an induction regimen for patients under 60 years; explicitly not supported by Evolent Policy due to lack of Level 1 evidence (randomized trials or meta-analyses) demonstrating superiority over Evolent-recommended alternatives.
Not supported
Single-agent gemtuzumab ozogamicin for induction or consolidation
Listed for patients age 60 years and older; explicitly not supported by Evolent Policy because Level 1 evidence is lacking to show superior outcomes compared to recommended alternatives.
Not supported

Background

Gemtuzumab ozogamicin (Mylotarg) is an anti‑CD33 antibody–drug conjugate indicated for treatment of CD33‑positive acute myeloid leukemia (AML). This policy governs authorization and medical‑necessity criteria for Mylotarg for Neighborhood Health Plan of Rhode Island members, including age‑based indications: newly diagnosed AML (age ≥1 month) and relapsed/refractory AML (age ≥2 years) provided the patient has not previously received Mylotarg. The policy also identifies specific regimens that are not supported due to lack of Level 1 evidence and defines documentation and dosing thresholds required for coverage.

Definitions

CD33-positive AML — definition (clarification)

Definition (repeat for clarity)CD33-positive AML: acute myeloid leukemia expressing the CD33 antigen; policy limits Mylotarg use to CD33-positive disease.

Age/setting summaryNewly diagnosed AML: age ≥1 month; Relapsed/refractory AML: age ≥2 years (must be Mylotarg-naïve).

Unsupported regimens noteCertain regimens containing Mylotarg are not supported (see policy for specific induction/consolidation regimens).

Revision History

2024-08-14Committee approval

Utilization Management Committee approved the Mylotarg (gemtuzumab ozogamicin) policy on August 14, 2024.

2024-08-30Effective dateLatest

Policy effective date established as August 30, 2024.

2017-09-13Committee review

Utilization Management Committee reviewed the policy (one of multiple historical committee review dates).