Overview

This document addresses the use of Mylotarg (gemtuzumab ozogamicin). Mylotarg is an antibody-drug conjugate composed of a monoclonal antibody targeting CD33 and the cytotoxic agent calicheamicin, which is released into the malignant cells upon binding. It is used to treat acute myeloid leukemia (AML). Originally FDA approved in 2000, Mylotarg was subsequently voluntarily withdrawn from the US market in 2010 due to safety and efficacy concerns. A required post-marketing study of the addition of Mylotarg to standard induction as first-line therapy for AML in individuals < 61 years of age showed significantly greater fatal induction toxicity and no improvement in survival compared to chemotherapy alone (NCT00085709; Petersdorf 2013). Re-approval for Mylotarg in 2017 was granted based on studies showing event-free survival advantage, overall survival advantage, or durable complete remission (Castaigne 2012, Amadori 2016, Hills 2014). The FDA approved indications include treatment of newly diagnosed AML (including induction and consolidation [post-remission] therapy) and treatment of relapsed or refractory AML.

The National Comprehensive Cancer Network® (NCCN) provides additional recommendations with a category 2A level of evidence for the use of Mylotarg. These include the use in high-risk acute promyelocytic leukemia (APL). NCCN identifies APL as high risk when white blood count (WBC) is greater than 10,000/mcL (cells per microliter). While NCCN includes a recommendation for Mylotarg in low-risk APL individuals if arsenic is not available or contraindicated, there is a lack of high-quality data to support this use.

Mylotarg has a black box warning for hepatotoxicity, including severe or fatal hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS) which has been reported within single agent and combination therapy. Individuals should be monitored frequently for signs and symptoms of VOD.

Definitions and Measures

• Acute promyelocytic leukemia (APL): an aggressive subtype of AML
• Anthracycline: A type of antibiotic that comes from certain types of Streptomyces bacteria and are used to treat many types of cancer. Anthracyclines damage the DNA in cancer cells, causing the cells to die.
• Monoclonal antibody: A protein developed in the laboratory that can locate and bind to specific substances in the body and on the surface of cancer cells.
• Refractory Disease: Illness or disease that does not respond to treatment.
• Relapse or recurrence: After a period of improvement, during which time a disease (for example, cancer) could not be detected, the return of signs and symptoms of illness or disease. For cancer, it may come back to the same place as the original (primary) tumor or to another place in the body.

Clinical Criteria

When a drug is being reviewed for coverage under a member’s medical benefit plan or is otherwise subject to clinical review (including prior authorization), the following criteria will be used to determine whether the drug meets any applicable medical necessity requirements for the intended/prescribed purpose.

Mylotarg (gemtuzumab ozogamicin)

Requests for Mylotarg (gemtuzumab ozogamicin) may be approved if the following criteria are met:

1. Individual has a diagnosis of CD33+ acute myeloid leukemia (AML); AND
2. Individual is using for one of the following:
   • As induction for AML; OR
   • As consolidation therapy for AML; OR
   • As treatment for relapsed or refractory AML;
   OR
3. Individual has a diagnosis of acute promyelocytic leukemia (APL) (NCCN 2A); AND
   • Individual has high-risk disease.

Requests for Mylotarg (gemtuzumab ozogamicin) may not be approved if the above criteria are not met and for all other indications.

Coding

The following codes for treatments and procedures applicable to this document are included below for informational purposes. Inclusion or exclusion of a procedure, diagnosis or device code(s) does not constitute or imply member coverage or provider reimbursement policy. Please refer to the member's contract benefits in effect at the time of service to determine coverage or non-coverage of these services as it applies to an individual member.

HCPCS

J9203
Injection, gemtuzumab ozogamicin, 0.1 mg [Mylotarg]

ICD-10 Diagnosis

• C92.00-C92.02
  Acute myeloblastic leukemia
• C92.40-C92.42
  Acute promyelocytic leukemia
• C92.50-C92.52
  Acute myelomonocytic leukemia
• C92.60-C92.62
  Acute myeloid leukemia with 11q23-abnormality
• C92.A0-C92.A2
  Acute myeloid leukemia with multilineage dysplasia
• C93.00-C93.02
  Acute monoblastic/monocytic leukemia
• C94.00-C94.02
  Acute erythroid leukemia
• C94.20-C94.22
  Acute megakaryoblastic leukemia

Document History

Revised: 02/24/2023