mhn Mylotarg (gemtuzumab ozogamicin) Prior Auth

Coverage Criteria for Gemtuzumab ozogamicin (Mylotarg)

Initial Therapy - Acute Myeloid Leukemia

Covered when ALL of the following are met for Initial Approval - Acute Myeloid Leukemia:

AML Initial Approval: 1. Diagnosis of CD33-positive AML; 2. Prescribed by or in consultation with an oncologist or hematologist; 3. Member meets (a or b): a. Age ≥ 1 month with newly diagnosed disease; b. Age ≥ 2 years with relapsed or refractory disease; 4. Mylotarg is prescribed as one of the following: a) As combination with chemotherapy for newly diagnosed disease: up to 5 doses; b) As single-agent therapy for newly diagnosed disease: up to 10 doses; c) As single-agent therapy for relapsed or refractory disease: up to 3 doses; d) As a component of repeating the initial successful induction regimen for relapsed or refractory disease if ≥ 12 months since induction regimen: up to 3 doses; 5. Request meets one of the regimen-specific dosing schemes or dose is supported by practice guidelines or peer-reviewed literature (prescriber must submit supporting evidence).

Prescribed regimen must be FDA-approved or recommended by NCCN; off-label dosing requires supporting evidence.

Initial Therapy - Acute Promyelocytic Leukemia (off-label)

Covered when ALL of the following are met for off‑label Acute Promyelocytic Leukemia:

APL Off‑label Initial Approval: 1. Diagnosis of acute promyelocytic leukemia; 2. Prescribed by or in consultation with an oncologist or hematologist; 3. Age ≥ 18 years; 4. Mylotarg is prescribed for no more than 10 doses total; 5. Dose is within FDA maximum limit for any FDA-approved indication or is supported by practice guidelines or peer-reviewed literature (prescriber must submit supporting evidence).

Prescribed regimen must be FDA-approved or recommended by NCCN; prescriber must submit supporting evidence for off‑label use.

Continuation Therapy

Covered when ALL of the following are met for Continued Therapy:

Continued Therapy: 1. Currently receiving medication via Centene benefit, or documentation supports current receipt and at least 30 days of therapy; 2. Member is responding positively to therapy; 3. For AML, member has NOT received the maximum recommended doses as described: a) combination with chemotherapy for newly diagnosed disease: up to 5 doses; b) single-agent therapy for newly diagnosed disease: up to 10 doses; c) single-agent therapy for relapsed or refractory disease: up to 3 doses; d) component of repeating initial successful induction regimen for relapsed/refractory disease if ≥12 months since induction: up to 3 doses; 4. For acute promyelocytic leukemia, member has not received ≥10 doses; 5. If request is for a dose increase, the requested dose must meet the regimen-specific dose limits (see pediatric and adult dosing schemes) or be supported by practice guidelines/peer-reviewed literature (prescriber must submit supporting evidence).

Prescribed regimen must be FDA-approved or recommended by NCCN; off-label dosing requires supporting evidence.

Requests for uses that are not FDA‑approved and that are not specifically addressed in this policy are excluded from coverage unless the provider supplies sufficient documentation of efficacy and safety in accordance with the applicable off‑label use policies (see CP.CPA.09 for Commercial, HIM.PA.154 for Health Insurance Marketplace, and CP.PMN.53 for Medicaid).

If state Medicaid coverage provisions conflict with this clinical policy, the state Medicaid coverage provisions take precedence. Providers should consult the applicable state Medicaid manual for specific coverage rules that may override the clinical policy.

Uses that are non‑FDA approved and that lack sufficient supporting evidence per the off‑label use policies are considered not authorized for coverage. Providers seeking coverage for off‑label uses must submit documentation meeting the standards in the referenced off‑label policies to be considered.

Covered Regimens and Dosing

Regimen	Dosing details	Indication / notes
Induction
3 mg/m2 IV on Days 1, 4, and 7 (up to 3 vials per induction regimen); maximum dose 4.5 mg per dose
Newly diagnosed acute myeloid leukemia in adults when given in combination with daunorubicin and cytarabine; if a second induction cycle is required, do NOT administer Mylotarg.

Regimen	Dosing details	Indication / notes
Induction
6 mg/m2 IV on Day 1 and 3 mg/m2 IV on Day 8; maximum induction dose 6 mg/m2 per dose
Newly diagnosed acute myeloid leukemia in adults as single‑agent therapy.

Regimen	Dosing details	Indication / notes
Single‑agent course
3 mg/m2 IV on Days 1, 4, and 7 for 1 cycle (up to 3 vials); maximum dose 4.5 mg per dose
Relapsed or refractory acute myeloid leukemia in patients age ≥ 2 years.

Regimen	Dosing details	Indication / notes
Combination with chemotherapy
Per NCCN-recommended combinations; specific dosing tied to the selected chemotherapy backbone and referenced NCCN guidance
Combination therapy options for newly diagnosed AML in adults and for relapsed/refractory AML per NCCN-supported off‑label use; prescriber should reference NCCN regimen selection.

Coding and Dose Limits

HCPCS codesHCPCS

J9203

Injection, gemtuzumab ozogamicin, 0.1 mg

Dose limits by age and regimen — examples and mappings

Pediatric induction (age 1 month to <18 years)Dose does not exceed 0.1 mg/kg if BSA <0.6 m2 or 3 mg/m2 if BSA ≥0.6 m2 (1 vial per induction cycle)

Pediatric intensificationDose does not exceed 0.1 mg/kg (BSA <0.6 m2) or 3 mg/m2 (BSA ≥0.6 m2) for 1 cycle (1 vial)

Adult combination induction (≥18 years)Induction: 3 mg/m2 IV on Days 1, 4, and 7 (up to 3 vials per induction)

Adult combination consolidationConsolidation: 3 mg/m2 IV on Day 1 of each consolidation cycle (up to 2 cycles; up to 1 vial per consolidation dose)

Adult single‑agent induction (≥18 years)Induction: 6 mg/m2 IV on Day 1 and 3 mg/m2 IV on Day 8 (maximum 6 mg/m2 on Day 1)

Adult single‑agent continuationContinuation: 2 mg/m2 IV on Day 1 every 4 weeks for up to 8 cycles

Provider Actions and Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required. The prescriber must submit documentation (such as office chart notes, lab results, or other clinical information) supporting that the member has met all approval criteria. Requests must meet the policy's initial approval criteria (including diagnosis of CD33-positive AML, prescriber specialty, age and regimen requirements) and prescribed regimen must be FDA-approved or recommended by NCCN; off-label dosing requires supporting literature or guideline evidence.

Prior authorization required for Gemtuzumab ozogamicin (Mylotarg).
Provider must supply supporting clinical documentation (office notes, labs, etc.) with the PA request.
Prescriber must be an oncologist or hematologist or consult with one per criteria.
Prescribed regimen must be FDA‑approved or recommended by NCCN; off‑label use must include supporting evidence.

Line of Therapy Guidance

first-line

Line specifics: Covers newly diagnosed (first-line) disease as combination with chemotherapy or single‑agent per specified regimens, and relapsed/refractory disease as indicated; specific dose and number of doses differ by line and age.

Biomarker Requirements

CD33 — biomarker requirement

Biomarker requirementCD33-positive

Role in coverageDiagnosis must be CD33-positive AML for initial approval under AML criteria

DocumentationPrescriber must document CD33-positive status as part of prior authorization submission

Definitions and References

AML — definition and coverage context

TermAcute myeloid leukemia (AML)

Coverage contextsCovered for newly diagnosed and relapsed/refractory CD33-positive AML per specified age- and regimen‑based dosing limits

Dose/line specificsCoverage includes combination induction/consolidation, single‑agent induction/continuation, and relapsed single‑agent regimens with defined maximum doses and number of doses

BSA — body surface area

TermBody surface area (BSA)

Use in dosingDosing thresholds reference BSA cutoffs (e.g., BSA <0.6 m2 vs ≥0.6 m2) to determine mg/kg vs mg/m2 dosing in pediatric patients

Background

Gemtuzumab ozogamicin (Mylotarg) is a CD33‑directed antibody–drug conjugate indicated for treatment of CD33‑positive acute myeloid leukemia (AML) and is the subject of this policy. The policy defines covered uses across newly diagnosed and relapsed/refractory AML for specified age groups, requires that the diagnosis be CD33‑positive, and specifies regimen‑ and age‑specific dosing and dose limits as conditions of coverage. Providers must follow the dosing schemes and approval durations described in the policy and submit prior authorization with supporting clinical documentation as required.

Provider Coding and Billing Note

Prior Authorization

Prior authorization and HCPCS J9203 (reminder)

Reiterating prior authorization and coding guidance: reference HCPCS J9203 when requesting authorization and note that authorization durations have been revised in plan reviews (Commercial: 6 months or to the member's renewal date).

HCPCS J9203 — Injection, gemtuzumab ozogamicin, 0.1 mg.
Commercial approval duration: '6 months or to the member's renewal date, whichever is longer.'

Revision History

2021-08-11annual_review

4Q 2021 annual review: updated age limit for acute promyelocytic leukemia per NCCN; updated dosing in section V; modified a reference to HIM.PA.154; for AML (age ≥18) revised induction vial quantity to 3 vials and removed maximum vial quantities for single-agent use.

2022-08-01annual_review

4Q 2022 annual review: removed maximum recommended number of doses from approval duration and clarified dosing within sections I/II; references reviewed and template changes applied.

2023-08-07annual_review

OpenPayer

Gemtuzumab Ozogamicin (Mylotarg)