Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions
Idhifa Approved for Certain Patients with AML
FDA news release; 2017 Aug 1
The FDA has approved Idhifa (enasidenib) for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) who have a specific genetic mutation.
Indications: Idhifa is an isocitrate dehydrogenase-2 inhibitor indicated for the treatment of adult patients with relapsed or refractory AML with an isocitrate dehydrogenase-2 mutation.
Dosage and administration: 100 mg orally once daily until disease progression or unacceptable toxicity.
Efficacy and safety: Approval is based on a single-arm trial showing that 19% of patients who took the drug experienced complete remission for a median 8.2 months, and 4% experienced complete remission with partial hematologic response for a median 9.6 months. Of the 157 patients who required transfusions of blood or platelets due to AML at the start of the study, ~one-third no longer required transfusions after treatment.
Side effects/risks: The most common adverse reactions are nausea, vomiting, diarrhea, elevated bilirubin, and decreased appetite.
FDA approves new targeted treatment for relapsed or refractory acute myeloid leukemia. [news release]. Silver Spring, MD: FDA. August 1, 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm569421.htm. Accessed August 7, 2017.
Idhifa [package insert]. Summit, NJ: Celgene Corporation. 2017. https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/209606s000lbl.pdf. Accessed August 7, 2017