CurrentMass General Brigham Health PlanPolicy N/A

Mylotarg (gemtuzumab ozogamicin) prior authorization and coverage

Defines prior authorization, coverage criteria, limits, and provider requirements for Mylotarg (gemtuzumab ozogamicin) for treatment of CD33‑positive acute myeloid leukemia (AML) under the plan.

Policy Summary

PayerMass General Brigham Health Plan

PolicyMylotarg (gemtuzumab ozogamicin) prior authorization and coverage

Policy CodePolicy N/A

Change TypeClinical and operational revisions

Effective DateJul 1, 2025

Next Review DateN/A

Key ActionObtain prior authorization and document oncologist/hematologist prescriber or provide consult notes; initial approvals differ by indication (3 cycles newly‑diagnosed; 1 cycle relapsed/refractory).

SourceLink

POLICY UPDATE CHANGES

Mylotarg has been removed from the pharmacy benefit and will be managed through the medical benefit only.

For newly diagnosed CD33‑positive AML, criteria will require either combination therapy or clinical rationale why combination is not appropriate or if member is ≥ 60 years of age.

Newly-dx & relapsedindications covered

3 cycles / 1 cycleinitial approval durations

≥1 mo / ≥2 yrsage thresholds

Medicalbenefit placement

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Coverage Criteria for Mylotarg (gemtuzumab ozogamicin)

Initial coverage criteria

Authorization may be granted when ALL of the following are met for each indication:

Newly-diagnosed CD33-positive AML (adults and pediatric ≥1 month): Member has newly-diagnosed CD33-positive acute myeloid leukemia; prescriber is an oncologist or hematologist or consult notes from an oncologist/hematologist are provided; appropriate dosing for the requested regimen is documented; the requested agent will be used in combination with cytarabine and daunorubicin or fludarabine OR there is a clinical rationale why combination therapy is not appropriate OR the member is > 60 years of age.

Relapsed or refractory CD33-positive AML (adults and pediatric ≥2 years): Diagnosis of relapsed or refractory CD33-positive acute myeloid leukemia; prescriber is an oncologist or hematologist or consult notes from an oncologist/hematologist are provided; appropriate dosing for the requested regimen is documented; member meets the age threshold (≥2 years); AND one of the following: documented relapsed or refractory AML OR prior therapy for AML with one systemic therapy (see Appendix for common regimens).

Duration and reauthorization limits

Limitations and reauthorization rules apply:

Initial approval duration: Newly-diagnosed CD33-positive AML: initial approval for three cycles; Relapsed or refractory CD33-positive AML (age ≥2 years): initial approval for one treatment cycle.

Reauthorization (newly-diagnosed monotherapy): For newly-diagnosed CD33-positive AML when used as monotherapy: reauthorization limited to a maximum of one induction and up to eight continuation cycles.

For members who are new to the plan and are currently receiving treatment with the requested medication, authorization may be reviewed on a case‑by‑case basis. Decisions should consider available documentation of prior therapy and clinical rationale; approvals are not automatically precluded by prior in‑flight treatment. The policy does not enumerate explicit exclusions tied to this situation, but absence of adequate case justification may affect the authorization outcome.

The document does not provide a list of conditions or scenarios that are explicitly labeled as not medically necessary. Instead, requests for members new to the plan who are already receiving the agent will be evaluated individually, and lack of supporting clinical documentation may lead to denial.

Regimen / Setting	Example agents & schedule (including gemtuzumab ozogamicin dosing options)
Patients < 60 years — standard induction	Cytarabine 1.5–3 g/m2 IV every 12 hours × 6 days; standard‑dose cytarabine with daunorubicin or idarubicin. Gemtuzumab ozogamicin may be given as a single 3 mg/m2 IV dose on day 1 (or alternatively day 2 or 3), or as three 3 mg/m2 doses on days 1, 4, and 7 when combined with standard‑dose cytarabine and anthracycline (CD33‑positive).
Standard‑dose cytarabine backbone (7+3)	Cytarabine 200 mg/m2 IV continuous infusion days 1–7 with daunorubicin 60–90 mg/m2 IV days 1–3 (or idarubicin 12 mg/m2 IV). For CD33‑positive disease, a single 3 mg/m2 IV dose of gemtuzumab ozogamicin may be given on day 1 (or day 3–4) or three doses on days 1, 4, and 7 as an option.
FLT3‑mutated induction	Standard‑dose cytarabine with daunorubicin plus oral midostaurin (e.g., midostaurin 50 mg PO every 12 hours on specified days). Gemtuzumab dosing options when added to standard cytarabine/daunorubicin regimens as above (single 3 mg/m2 or three‑dose schedules) for CD33‑positive AML.
Liposomal dual‑drug induction	Dual‑drug liposomal encapsulation of cytarabine and daunorubicin (e.g., daunorubicin 44 mg/m2 and cytarabine 100 mg/m2 on days 1, 3, and 5). Gemtuzumab may be used in CD33‑positive patients per prescriber decision; dosing examples in document are single 3 mg/m2 or three 3 mg/m2 doses when combined with cytarabine/daunorubicin backbones.
Patients ≥60 years / lower‑intensity regimens	Low‑intensity options include azacitidine or decitabine alone, or venetoclax combinations (venetoclax daily with decitabine 20 mg/m2 days 1–5 or azacitidine 75 mg/m2 days 1–7). For CD33‑positive older patients, gemtuzumab may be considered as monotherapy or in combination with standard or lower‑intensity backbones per clinical rationale and prescriber judgment; documented gemtuzumab schedules include single 3 mg/m2 dose or three doses (days 1, 4, 7).
Venetoclax‑based regimens	Venetoclax once daily PO combined with decitabine 20 mg/m2 IV days 1–5 or azacitidine 75 mg/m2 IV days 1–7, or low‑dose cytarabine 20 mg/m2 days 1–10. When used in CD33‑positive patients, gemtuzumab dosing examples in the document include a single 3 mg/m2 IV dose given on day 1 (or alternative single‑day timing) or three 3 mg/m2 doses on days 1, 4, and 7.

Coding and Age Thresholds

Codes mentioned/placeholdermixed

affected codes

No specific CPT/HCPCS/NDC codes listed in document; refer to medical benefit billing.

Age thresholds

Newly-diagnosed (age threshold)≥ 1 month (pediatric and adult) for newly‑diagnosed CD33‑positive AML

Relapsed/Refractory (age threshold)≥ 2 years for relapsed or refractory CD33‑positive AML

Consideration for monotherapyMember ≥ 60 years — may be considered for monotherapy instead of combination therapy

Prior Authorization, Documentation, and Provider Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required. Authorization may be reviewed on a case-by-case basis for members new to the plan who are currently receiving treatment; provide clinical justification and documentation when historical treatment records are unavailable.

Prior authorization required
Case-by-case review for members new to the plan currently receiving treatment — document justification if prior history is unavailable

Documentation Required

Specialist Prescriber or Consult Notes Required

Prescriber must be a hematologist or oncologist. If the ordering provider is not a hematologist or oncologist, submit consult notes from a hematologist or oncologist with the prior authorization request.

Prescriber must be an oncologist or hematologist
If prescriber is not a specialist, include consult notes from an oncologist or hematologist

First-line and Salvage Therapy

first-line | salvage

Line of therapy: Newly diagnosed (induction/combination or monotherapy with documented clinical rationale) OR relapsed/refractory (salvage; monotherapy allowed with prior therapy documentation as applicable).

CD33 Biomarker Requirements

CD33 (biomarker requirement)

Biomarker requirementCD33 positivity is required — therapy is indicated for CD33‑positive acute myeloid leukemia

Applies to indicationsRequired for both newly‑diagnosed and relapsed/refractory AML indications

VerificationPrescribing and authorization criteria reference CD33‑positive disease to establish eligibility

Key Definitions

CD33‑positive AML — definition and target population

DefinitionAcute myeloid leukemia expressing the CD33 antigen — the target population for gemtuzumab ozogamicin (Mylotarg)

Indicated populationsNewly‑diagnosed CD33‑positive AML (adults and pediatric ≥1 month) and relapsed/refractory CD33‑positive AML (adults and pediatric ≥2 years)

Therapeutic roleGemtuzumab ozogamicin is a CD33‑directed antibody‑drug conjugate used as combination therapy or monotherapy per indication and clinical rationale

Background on Mylotarg

Mylotarg (gemtuzumab ozogamicin) is a CD33‑directed antibody‑drug conjugate (ADC) indicated for the treatment of CD33‑positive acute myeloid leukemia (AML). It is approved for use in newly diagnosed disease (adults and pediatric patients ≥1 month of age) as monotherapy or in combination with standard induction regimens, and for relapsed or refractory disease (adults and pediatric patients ≥2 years of age) as monotherapy. The policy references standard AML induction regimens and compendia/prescribing information to guide appropriate combination and dosing strategies.