MedPath

Enasidenib

Generic Name
Enasidenib
Brand Names
Idhifa
Drug Type
Small Molecule
Chemical Formula
C19H17F6N7O
CAS Number
1446502-11-9
Unique Ingredient Identifier
3T1SS4E7AG
Background

Enasidenib is an orally available treatment for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with specific mutations in the isocitrate dehydrogenase 2 (IDH2) gene, which is a recurrent mutation detected in 12-20% of adult patients with AML . Patients eligible for this treatment are selected by testing the presence of IDH2 mutations in the blood or bone marrow. This small molecule acts as an allosteric inhibitor of mutant IDH2 enzyme to prevent cell growth, and it also has shown to block several other enzymes that play a role in abnormal cell differentiation. First developed by Agios Pharmaceuticals and licensed to Celgene, enasidenib was approved by U.S. Food and Drug Administration on August 1, 2017.

Indication

Indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase-2 (IDH2) mutation.

Associated Conditions
Refractory Acute Myeloid Leukemia (AML), Relapsed Acute Myelogenous Leukemia (AML)

Luspatercept Plus Lenalidomide Shows Promise in Non-del(5q) Myelodysplastic Syndrome

• A phase 1b trial combining luspatercept and lenalidomide demonstrates safety and preliminary efficacy in patients with non-del(5q) myelodysplastic syndrome (MDS). • The combination therapy showed a 50% hematologic improvement rate among evaluable patients, with notable red blood cell transfusion independence. • The study established a recommended phase 2 dose (RP2D) of lenalidomide at 10 mg daily and luspatercept at 1.0 mg/kg every 21 days. • These findings support further investigation of this combination to improve outcomes for lower-risk MDS patients lacking the del(5q) abnormality.

IDHIFA Clinical Trial Induces Remission in AML Patient with IDH2 Mutation

• A 33-year-old patient with acute myeloid leukemia (AML) achieved remission through a clinical trial using IDHIFA, an oral medication targeting the IDH2 mutation. • The patient, who initially underwent intensive remission induction therapy with chemotherapy, opted for the IDHIFA trial as an alternative to a bone marrow transplant. • IDHIFA works by turning off the mutated IDH2 gene, restoring normal cell function and offering a targeted approach to treating AML in patients with this specific mutation. • The patient experienced minimal side effects and has returned to work and normal activities, highlighting the potential of targeted gene therapy in leukemia treatment.

FDA Approves Pyrukynd as First Treatment for Pyruvate Kinase Deficiency

• The FDA has approved mitapivat (Pyrukynd) as the first treatment for adults with pyruvate kinase (PK) deficiency, a rare inherited anemia. • Pyrukynd, a small molecule, works by activating PK enzymes to improve red blood cell function, offering a new approach to managing this condition. • Clinical trials demonstrated that Pyrukynd significantly increased hemoglobin levels in non-transfusion-dependent patients and reduced transfusion burden in regularly transfused patients. • Agios Pharmaceuticals, now focused on rare genetic diseases, estimates that many PK deficiency cases remain undiagnosed, highlighting the need for increased awareness and testing.
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