FDA Approves Idhifa (Enasidenib) for Acute Myeloid Leukemia

Written By:Elizabeth Mendes

The US Food and Drug Administration (FDA) has approved the use of the targeted therapy drug Idhifa (enasidenib) to treat adults who have a specific type of acute myeloid leukemia (AML). The drug is for AML patients who have a mutation in a gene called IDH2. The FDA also approved a test to check for the mutation. Idhifa is intended to treat AML patients whose cancer has either not responded to or has come back after prior treatment.  

Idhifa works by blocking certain proteins on cancer cells that help them grow.

The FDA based its approval on a clinical trial of 199 people with AML whose cancer had come back or never responded to previous treatment. With a minimum of 6 months of treatment with Idhifa, 19% of patients experienced complete remission for a median 8.2 months.

Idhifa was evaluated through the FDA’s priority review program, which is meant to shorten the FDA’s decision timeline to less than 6 months, from the typical 10 months. The drug also received orphan product designation, which provides incentives to the drug’s developers because it is intended to treat a rare disease.

Idhifa is taken as a pill, once a day. The most common side effects of the drug are nausea, vomiting, diarrhea, increased levels of bilirubin (a substance found in bile) and decreased appetite. 

Women who are pregnant or breastfeeding should not take Idhifa because it may cause harm to a developing fetus or a newborn baby.

Idhifa is marketed by Celgene Corporation, and the RealTime IDH2 Assay is marketed by Abbott Laboratories.

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