Enasidenib
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| ID | DRUG-ENASIDENIB |
|---|---|
| Tipo | Fármaco |
| Alegais | IdhifaЕнасиденіб |
| Estado | reviewed 2026-04-30 | pending_clinical_signoff |
| Doenças | DIS-AML |
| Fontes | SRC-ELN-AML-2022 SRC-NCCN-AML-2025 |
Fatos sobre as drogas
| Classe | Inibidor seletivo da enzima IDH2 (mutante) |
|---|---|
| Mecanismo | Small-molecule allosteric inhibitor of mutant IDH2 enzyme (R140Q, R172K hotspots). Mutant IDH2 catalyzes neomorphic conversion of α-ketoglutarate to 2-hydroxyglutarate (2-HG), an oncometabolite that drives DNA + histone hypermethylation and blocks myeloid differentiation. Enasidenib reduces 2-HG, restoring TET2-dependent demethylation and inducing terminal differentiation of leukemic blasts. FDA-approved Aug 2017 on the basis of phase 1/2 single-arm data in R/R AML (AG221-AML-001). |
| Dose típica | 100 mg PO once daily, continuous, until disease progression or unacceptable toxicity. Take with or without food at the same time each day. Median time to first response ~1.9 months; continue ≥6 months before declaring non-response. |
| Ucrânia registada | Falso |
| NSZU reembolsado | Falso |
| Ucrânia verificada pela última vez | 2026-04-30 |
Avisos
- Síndrome de diferenciação — fatal se não reconhecido; corticosteróides + hidroxiureia + interrupção da dose obrigatório (~14% em IDHENTIFY)
Notas
Pivotal trials: AG221-AML-001 (Stein et al., Blood 2017) — phase 1/2 monotherapy in R/R IDH2-mut AML: ORR 38%, CR 19%, median OS 9.3 mo. IDHENTIFY (NCT02577406) — phase 3 enasidenib mono vs conventional care in R/R AML; did not show OS benefit overall but enriched benefit in IDH2-mut HMA-naive subgroup. AG221-AML-005 — combination with azacitidine in 1L unfit IDH2-mut AML: ORR 71%, CR 53% — basis for emerging 1L combination use. Differentiation syndrome (~14%) is the signature AE — same monitoring + dexamethasone protocol as ivosidenib. Hyperbilirubinemia (~80% any grade) is biochemical UGT1A1 inhibition — usually asymptomatic, no dose adjustment unless symptomatic. Ukraine: NOT registered, NOT reimbursed; EU access also limited (CHMP negative). Bibliographic record SRC-IDHENTIFY is available; clinical claim linkage requires dual clinical sign-off.
Usado por
Esquema terapêutico
REG-ENASIDENIB-AML- Enasidenib monotherapy for R/R IDH2-mutated AML