Idhifa Approved for Relapsed/Refractory AML with IDH2 Mutation

Idhifa is an isocitrate dehydrogenase-2 inhibitor
Idhifa is an isocitrate dehydrogenase-2 inhibitor

The Food and Drug Administration (FDA) has approved Idhifa (enasidenib; Celgene) for the treatment of adults with relapsed or refractory acute myeloid leukemia (AML) with a specific genetic mutation. Idhifa is approved for use with the Real Time IDH2 Assay companion diagnostic (Abbott), intended to identify specific mutations in the IDH2 gene in these patients. 

“AML is a complex, heterogeneous disease, which is particularly difficult to treat in the relapsed or refractory setting,” said Martin Tallman, M.D., Hematologic Oncologist and Chief, Leukemia Service at Memorial Sloan Kettering Cancer Center. “IDH2 mutations inhibit the normal maturation of myeloid cells, so having a treatment that targets this mechanism is promising for patients and encouraging to us as physicians who have it as our goal to provide options for every patient.”

Idhifa, an isocitrate dehydrogenase-2 inhibitor, was evaluated in a single-arm trial (n=199) of patients with relapsed or refractory AML who had IDH2 mutations. The study assessed the percentage of patients with complete remission (no evidence of disease and full recovery of blood counts post-treatment) as well as patients with complete remission with partial hematologic recovery. 

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After a minimum 6 months of treatment, complete remission was seen in 19% of patients for a median of 8.2 months. Complete remission with partial hematologic recovery was seen in 4% of patients with a median of 9.6 months. Following treatment with Idhifa, 34% of patients who required blood or platelet transfusions at the start of study no longer required them. 

Nausea, vomiting, increased bilirubin, and reduced appetite were reported as common adverse effects of Idhifa. The drug carries a Boxed Warning regarding the risk of differentiation syndrome, which can be fatal if untreated. Symptoms may include fever, dyspnea, acute respiratory distress, radiographic pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain, peripheral edema, or hepatic/renal or multi-organ dysfunction. 

Idhifa was previously granted FDA Priority Review and Orphan Drug designations. The product will be available as 50mg and 100mg strength tablets in 30-count bottles.

For more information visit FDA.gov.