Acute Myeloid Leukemia | Decision Base | US/EU | 2014

With Few Good Treatment Choices, Which Emerging Therapies Hold the Greatest Potential for Managing AML and What Types of Therapies Will Receive Reimbursement?

Despite the introduction and use of hypomethylating agents for acute myeloid leukemia (AML), treatment of this disease has changed little over the past several decades. Withdrawal of the only FDA-approved monoclonal antibody for AML—Pfizer’s Mylotarg (gemtuzumab ozogamicin)—from the U.S. market in 2010 was not only met with disappointment and controversy, but it left clinicians with fewer treatment choices. Given patients’ short survival times, the level of unmet need remains high, particularly for elderly patients, who may not be eligible for intensive induction chemotherapy. Even though regulatory incentives such as orphan drug designation and breakthrough therapy designation are available to developers of therapies for AML, which is a rare cancer, these incentives have not been enough to attract substantial levels of clinical development activity in AML. Although several therapies being investigated for AML will offer new treatment options for elderly AML patients with newly diagnosed disease, opportunity remains high for therapies that extend survival and slow disease progression.

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