Acute myeloid leukemia

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At a Glance

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Regulatory Watchboard

10 events
Apr 2026VANFLYTA: New indication approved
FDAcompleted
Jan 2026VYXEOS: New indication approved
FDAcompleted
Nov 2025

Komzifti: FDA approved

treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation who have no satisfactory alternative treatment options

FDAcompleted
Jan 2025Gut Health in Children With Cancer

Great Ormond Street Hospital for Children NHS Foundation Trust

TrialRECRUITING
Jan 2025Avapritinib Combined With Azacitidine and Venetoclax in the Treatment of Relapsed AML After Allo-HSCT

Institute of Hematology & Blood Diseases Hospital, China — PHASE2

TrialRECRUITING
Dec 2024Testing the Combination of an Anti-Cancer Drug, Iadademstat, With Other Anti-Cancer Drugs (Venetoclax and Azacitidine) for Treating AML

National Cancer Institute (NCI) — PHASE1

TrialRECRUITING
Sep 2024Interferon-γ (IFN-γ) With Donor Leukocyte Infusion to Treat Relapsed Acute Myeloid Leukemia and Myelodysplastic Syndromes Post Allogeneic Hematopoietic Stem Cell Transplantation

Sawa Ito, MD — PHASE2

TrialRECRUITING
Jul 2023

Vanflyta: FDA approved

in combination with standard cytarabine and anthracycline induction and cytarabine consolidation, and as maintenance monotherapy following consolidation chemotherapy, for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3 internal tandem duplication (ITD)-positive as detected by an FDA-approved test

FDAcompleted
May 2023Allo HSCT Using RIC and PTCy for Hematological Diseases

Masonic Cancer Center, University of Minnesota — PHASE2

TrialRECRUITING
Dec 2022

Rezlidhia: FDA approved

treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test

FDAcompleted

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

9 programs

FINANCIAL LANDSCAPE SUMMARY

9

Total programs

9

Open now

9

Copay cards

Copay Assistance9

REZLIDHIA

Rigel

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Idhifa

Servier Pharmaceuticals

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VANFLYTA

Daiichi Sankyo

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RYDAPT

Novartis

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VYXEOS

Jazz

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GRAFAPEX

Unknown

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Idamycin PFS

Pfizer

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Daurismo

Pfizer

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VYXEOS ; Vyxeos ; Vyxeos

Celator Pharmaceuticals, Inc. (a Jazz Pharmaceuticals Company)

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Approved Treatments

8 FDA-approved

Komzifti

(ziftomenib)Orphan drug

Kura Oncology, Inc.

Menin Inhibitor [EPC]

12.1 Mechanism of Action Ziftomenib is a menin inhibitor that blocks the interaction of menin and lysine [K]-specific methyltransferase 2A (KMT2A). Ac...

Approved Nov 2025FDA label ↗

Vanflyta

(quizartinib)Orphan drug

Daiichi Sankyo, Inc.

12.1 Mechanism of Action Quizartinib is a small molecule inhibitor of the receptor tyrosine kinase FLT3. Quizartinib and its major active metabolite A...

Approved Jul 2023FDA label ↗

Rezlidhia

(olutasidenib)Orphan drug

Rigel Pharmaceuticals, Inc.

12.1 Mechanism of Action Olutasidenib is a small-molecule inhibitor of mutated isocitrate dehydrogenase-1 (IDH1). In patients with AML, susceptible ID...

Approved Dec 2022FDA label ↗

VYXEOS

(Cytarabine:daunorubicin liposome injection; daunorubicin and cytarabine (liposome); daunorubicin and cytarabine liposome for injection)Orphan drug

Celator Pharmaceuticals, Inc. (a Jazz Pharmaceuticals Company)

Approved Mar 2021FDA label ↗

Mylotarg

(Gemtuzumab ozogamicin)Orphan drug

Wyeth Pharmaceuticals, Inc., a Pfizer Company

CD33-directed Immunoconjugate [EPC]

12.1 Mechanism of Action Gemtuzumab ozogamicin is a CD33-directed antibody-drug conjugate (ADC). The antibody portion (hP67.6) recognizes human CD33 a...

Approved Sep 2017FDA label ↗

Idhifa

(enasidenib)Orphan drug

Celgene Corporation

12.1 Mechanism of Action Enasidenib is a small molecule inhibitor of the isocitrate dehydrogenase 2 (IDH2) enzyme. Enasidenib targets the mutant IDH2 ...

Approved Aug 2017FDA label ↗

Rydapt

(RYDAPT)Orphan drug

Novartis Pharmaceuticals Corporation

Kinase Inhibitor [EPC]

12.1 Mechanism of Action Midostaurin is a small molecule that inhibits multiple receptor tyrosine kinases. In vitro biochemical or cellular assays hav...

Approved Apr 2017FDA label ↗

VENCLEXTA

(venetoclax)Orphan drugstandard

AbbVie, Inc

BCL-2 Inhibitor [EPC]

12.1 Mechanism of Action Venetoclax is a selective and orally bioavailable small-molecule inhibitor of BCL-2, an anti-apoptotic protein. Overexpressio...

FDA label ↗

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Clinical Trial Landscape

7 active trials
3Phase 2
2Phase 1
2Unknown
7Total recruiting
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Recent News & Research

6 articles
The Journal of biological chemistryApr 18, 2026

Mesothelin promotes acute myeloid leukemia progression through LYN-dependent signaling.

Mesothelin (MSLN) is a glycosylphosphatidylinositol-anchored cell surface protein that is overexpressed in several solid tumors and in one-third of pediatric acute myeloid leukemia (AML) cases. It rep...

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Annals of hematologyApr 17, 2026

CLL-1: An emerging target for immunotherapy in acute myeloid leukemia.

AML is an aggressive haematological malignancy characterised by uncontrolled proliferation and differentiation arrest of myeloid progenitor/stem cells. Conventional treatment methods principally entai...

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Leukemia researchApr 16, 2026

The emerging role of plasmacytoid dendritic cell proliferation in acute myeloid leukemia.

The mature plasmacytoid dendritic cell proliferation associated with myeloid neoplasm represents a clonal proliferation of plasmacytoid dendritic cells within myeloproliferative and myelodysplastic di...

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Journal of the National Cancer InstituteApr 9, 2026

Real-world generalizability of clinical trial cytomolecular risk in pediatric acute myeloid leukemia: a report from the REAL-AML cohort.

Cytomolecular features critical for risk-stratified treatment determination in pediatric acute myeloid leukemia (AML) were expanded in Children's Oncology Group (COG) Phase III trial AAML1831 based on...

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Molecular therapy : the journal of the American Society of Gene TherapyApr 2, 2026

CD96 as a therapeutic target for CAR T cell therapy in acute myeloid leukemia.

Therapies leveraging chimeric antigen receptor (CAR) T cells for acute myeloid leukemia (AML) are limited by the scarcity of leukemia stem cell (LSC)-specific antigens. Here, we found that CD96 is exp...

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GenesMar 28, 2026

The Exosome Landscape in Acute Myeloid Leukemia: From Molecular Mechanisms to Translational Frontiers.

Acute myeloid leukemia (AML) is a biologically heterogeneous hematologic malignancy arising from the oncogenic transformation of hematopoietic stem and progenitor cells, resulting in clonal expansion ...

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Specialist Network

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