Appeal type: Experimental/Investigational
Decision: Overturned

Diagnosis: Acute Myeloid Leukemia.
Treatment: Xospata 40 MG OR TABS
The insurer denied Xospata 40 mg OR TABS.
The determination is overturned.

The patient has a history of acute myeloid leukemia (AML) with FLT-3 internal tandem duplication (ITD). He has been receiving chemotherapy as treatment for his condition and has completed only 4 of 5 intended cycles of chemotherapy which increases his risk treatment failure. The provider is recommended treatment with an agent that targets FLT3, but the only pediatric approved agent, midostaurin, is not an appropriate treatment for this patient. Midostaurin has a history of causing cardiac rhythm abnormalities and this patient has had prolonged QTc interval on this medication. The provider is now recommending treatment with Xospata. At issue is whether this is likely to be more beneficial than any standard treatment or treatments for the insured's life threatening or disabling condition or disease.
The proposed health service or treatment Xospata 40 milligrams (mg) OR tabs is likely to be more beneficial than any standard treatment or treatments for the insured's life threatening or disabling condition or disease.
This patient has high-risk acute myeloid leukemia (AML) for which the provider has recommended the use of Xospata as part of a potential maintenance treatment plan. This product was previous denied as it was not Food and Drug Administration (FDA) approved for this indication and is specifically not considered to be proven in the setting of maintenance therapy for AML. The patient is also not a candidate for midostaurin due to potential for cardiac conduction concerns, so Xospata is considered to be the most appopriate treatment at this time.
As per the National Comprehensive Cancer Network (NCCN), for FLT3-mutated acute myeloid leukemia (AML) some different types of chemotherapy regimens are typically considered appropriate. The National Comprehensive Cancer Network (NCCN) states that the regimen for midostaurin is typically recommended for this disease subtype and while midostaurin is not FDA approved for maintenance therapy, this product was to be used in study design for consolidation and maintenance phase of therapy for a total of 12 months. The median time receiving maintenance therapy was 9 months after allogeneic transplant and 10.5 months after high-dose chemotherapy consolidation.
One of the big issues in this clinical case is the potential use of the requested product as a maintenance therapy for FLT3 positive AML. A review posted last month demonstrates why maintenance therapy is important in this group of patients and why its use is being proposed. Midostaurin and gilteritinib are both being considered for maintenance treatment and the overall survival benefit. Gilteritinib is being evaluated in a placebo-controlled phase III protocol where patients are randomized to this treatment during induction / consolidation and for 12 months as maintenance therapy. As this patient cannot take the FDA-approved product for this diagnosis and given the need for some sort of additional treatment since he could not complete the standard treatment regimen, Xospata is the most appropriate treatment option going forward.
Therefore, based on the clinical information provided for review and the current peer-reviewed literature / standards of care in the literature, the use of Xospata 40 mg OR tabs is the best available treatment option for this patient's life-threatening disease.