Idhifa Approved for Acute Myeloid Leukemia
August 1, 2017 – The U.S. FDA has approved Idhifa (enasidenib), manufactured by Celgene Corporation, for treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) who have an IDH2 gene mutation. Idhifa is approved for use in conjunction with a companion diagnostic, the RealTime IDH2 Assay, which identifies specific mutations in the IDH2 gene in patients who have AML. An isocitrate dehydrogenase-2 inhibitor, Idhifa blocks several enzymes that promote cancerous cell growth. Patients treated with Idhifa in a clinical trial experienced a reduction in the need for red cell and platelet transfusions, and in some cases achieved complete remission.
AML forms in the bone marrow and progresses rapidly, causing an increased number of abnormal white blood cells in the bloodstream and bone marrow. Approximately 21,380 patients will be diagnosed with AML in 2017, according to estimates from the National Cancer Institute at the National Institutes of Health. An estimated 10,590 patients will die of AML this year alone.
Idhifa will be available in both 50mg tablets and 100mg tablets. Recommended dosing is one 100mg tablet taken once daily, with or without food, until the patient’s disease progresses, or the patient experiences unacceptable toxicity. For patients who do not experience disease progression or unacceptable toxicity, treatment should continue for a minimum of six months to allow time for a clinical response. Side effects can be managed in some cases by reducing the dosage to 50mg per day.
Common side effects of Idhifa include nausea, vomiting, diarrhea, increased levels of bilirubin (a substance found in bile), and decreased appetite. Idhifa carries a black box warning for differentiation syndrome, which is associated with rapid proliferation and differentiation of myeloid cells, and can be fatal if left untreated. Signs and symptoms include fever, difficulty breathing, acute respiratory distress, inflammation of the lungs, fluid around the lungs or heart, rapid weight gain, swelling, or liver, kidney, or multi-organ dysfunction. Doctors should treat patients with corticosteroids at the first suspicion of differentiation syndrome, and monitor these patients closely.
Adverse reactions or quality problems experienced with the use of a recalled product may be reported to the FDA’s MedWatch Adverse Event Reporting Program either online, by U.S. mail or by fax.
- Complete and submit the report Online: www.fda.gov/medwatch/report.htm
- U.S. Mail or Fax: Download form www.fda.gov/MedWatch/getforms.htm or call 1-800-332-1088 to request a reporting form, then complete and return to the address on the pre-addressed form, or submit by fax to 1-800-FDA-0178
Actual drug patent expiration dates and availability of new medications are subject to change due to patent litigation, settlement agreements, additional patents, exclusivities, and final FDA approval. Distribution and availability of new medications at pharmacies may not occur immediately following FDA approval. Patients are advised to speak with their healthcare professional or pharmacist regarding appropriateness as well as actual availability.
*This is provided for information only. The reference to any medication above does not mean the medication is covered by your plan.