Idhifa for MDS – pro
IDHIFA® (enasidenib) is indicated for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA-approved test. The IDH2 inhibitor enasidenib (Idhifa) demonstrated promising efficacy in combination with azacitidine and as a single agent in patients with high-risk, IDH2-mutated myelodysplastic syndrome (MDS), according to findings from a phase 2 study (NCT03383575) that were presented during the 2021 ASCO Annual Meeting and subsequently published in the Journal of Clinical Oncology.
Another recent 2-arm multicenter study of 50 patients was published in 2023 by DiNardo et al. It found that Enasidenib is an effective treatment option for mIDH2 MDS, both in combination with azacitidine for treatment-naïve high-risk MDS, and as a single agent after prior HMA therapy.
S, DiNardo CD, Takahashi K, et al. Phase II study of the IDH2-inhibitor enasidenib in patients with high-risk IDH2-mutated myelodysplastic syndrome (MDS). J Clin Oncol. 2021;39(suppl 15):7010.
DiNardo CD, Venugopal S, Lachowiez C, Takahashi K, Loghavi S, Montalban-Bravo G, Wang X, Carraway H, Sekeres M, Sukkur A, Hammond D, Chien K, Maiti A, Masarova L, Sasaki K, Alvarado Y, Kadia T, Short NJ
Idhifa Prescribing Information 2024
NCCN MDS 2024, Daver N, Borthakur G, Ravandi F, Kantarjian HM, Patel B, Dezern A, Roboz G, Garcia-Manero G. Targeted therapy with the mutant IDH2 inhibitor enasidenib for high-risk IDH2-mutant myelodysplastic syndrome. Blood Adv. 2023 Jun 13;7(11):2378-2387.