Tag: Acute Myeloid Leukemia

FDA Approves All-Oral Treatment for Newly Diagnosed Acute Myeloid Leukemia

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SUMMARY: The FDA on May 13, 2026, approved an oral combination of Decitabine and Cedazuridine tablets (INQOVI®) with Venetoclax for the treatment of newly diagnosed Acute Myeloid Leukemia (AML) in adults 75 years or older, or who have comorbidities that preclude the use of intensive induction chemotherapy.

The American Cancer Society estimates that in 2026, 22,720 new cases of Acute Myeloid Leukemia (AML) will be diagnosed in the United States and 11,500 patients will die of the disease. AML is one of the most common types of leukemia in adults and can be considered as a group of molecularly heterogeneous diseases with different clinical behavior and outcomes. A significant percentage of patients with newly diagnosed AML are not candidates for intensive chemotherapy or have disease that is refractory to standard chemotherapy.

Advancing Convenience Without Compromising Efficacy

The treatment landscape for older adults with AML who are not candidates for intensive induction chemotherapy has evolved significantly with the introduction of Venetoclax-based combinations. However, despite improved outcomes, current standards of care still rely heavily on parenteral HypoMethylating Agents (HMAs), requiring frequent visits to healthcare facilities and creating substantial logistical and quality-of-life challenges for patients and caregivers.

Findings from the Phase 1–2 ASCERTAIN-V trial offer compelling evidence that an entirely oral regimen consisting of Decitabine–Cedazuridine (INQOVI®) and Venetoclax (VENCLEXTA®) may provide a clinically effective alternative, while reducing the treatment burden associated with injectable therapies. The study evaluated the safety, pharmacokinetics, and efficacy of this oral combination in patients with newly diagnosed AML who were either 75 years of age or older or considered unsuitable for intensive chemotherapy due to significant comorbidities.

The Rationale for an All-Oral Approach

Venetoclax combined with Azacitidine or Decitabine has become the established frontline treatment for patients with AML who are ineligible for intensive chemotherapy. While these regimens have demonstrated meaningful survival benefits, they require repeated administration of intravenous or subcutaneous HMAs over several days during each treatment cycle.

Decitabine–Cedazuridine was developed to address this challenge by delivering oral Decitabine exposure comparable to IV administration. Cedazuridine inhibits cytidine deaminase, preventing premature degradation of Decitabine in the gastrointestinal tract and allowing therapeutic systemic exposure through oral dosing. Previous studies have demonstrated pharmacokinetic equivalence between oral Decitabine–Cedazuridine and intravenous Decitabine, paving the way for evaluation in AML.

The ASCERTAIN-V investigators sought to determine whether combining oral Decitabine–Cedazuridine with oral Venetoclax could maintain the efficacy expected from HMA-Venetoclax therapy while improving treatment convenience and potentially enhancing patient experience.

Study Design and Patient Population

ASCERTAIN-V was a multicenter, open-label, nonrandomized Phase 1–2 study conducted across 34 academic and community treatment centers in the United States, Canada, and Spain.

The trial enrolled patients with newly diagnosed AML who met one of two criteria:

  • Age 75 years or older, or
  • Younger patients with significant comorbidities that precluded intensive chemotherapy.

Qualifying comorbid conditions included severe cardiac or pulmonary disease, impaired renal function, moderate hepatic dysfunction, or reduced performance status.

All participants received:

  • Oral Decitabine–Cedazuridine (35 mg/100 mg) on days 1–5 of each 28-day cycle
  • Oral Venetoclax, initiated with a standard ramp-up schedule and continued at 400 mg daily

A total of 189 patients were enrolled across the study, including 101 patients in the pivotal Phase 2b cohort that served as the primary efficacy population.

Pharmacokinetic Findings Confirm Combination Feasibility

A key objective of the study was to determine whether coadministration of Venetoclax altered exposure to Decitabine–Cedazuridine or vice versa.

Results demonstrated no clinically meaningful drug–drug interactions between the two agents. Pharmacokinetic analyses showed that systemic Decitabine exposure remained consistent with previous studies evaluating oral Decitabine–Cedazuridine alone, supporting the feasibility of combining the two oral therapies without compromising drug delivery. These findings are particularly important because maintaining predictable drug exposure is essential when replacing established IV regimens with oral alternatives.

Encouraging Clinical Activity in a High-Risk Population

The pivotal Phase 2b cohort met its predefined efficacy objective and demonstrated substantial antileukemic activity.

Among evaluable patients:

  • Complete remission (CR) was achieved in 47% of patients.
  • The combined rate of Complete Remission and Complete Remission with incomplete hematologic recovery (CR + CRi) reached 63%.
  • Median Overall Survival was 15.5 months.
  • Among patients achieving Complete Remission, 75% maintained their response at 12 months.

Minimal Residual Disease (MRD) analyses, although exploratory and not standardized, also provided encouraging signals. More than half of responding patients achieved MRD negativity by multiparameter flow cytometry, a finding often associated with deeper remissions and improved long-term outcomes.

Managing Myelosuppression Through Adaptive Treatment Strategies

As expected with Venetoclax-based AML therapy, myelosuppression remained the principal toxicity observed during treatment.

The most common grade 3 or higher adverse events in the pivotal phase 2b cohort included Anemia (30%), Neutropenia (26%) and Febrile neutropenia (25%). Early mortality remained within expected ranges for this older and medically complex population, with 30-day and 60-day mortality rates of 3% and 10%, respectively.

Importantly, the investigators incorporated treatment modifications during the Phase 2b portion of the study to reduce prolonged cytopenias. Early bone marrow assessments were used to confirm blast clearance before completion of a full 28-day course of Venetoclax. Once remission was documented, Venetoclax exposure was shortened and treatment schedules for both agents were adjusted to facilitate hematologic recovery.

This strategy appeared successful. As treatment progressed, the duration of Venetoclax administration decreased, accompanied by reductions in serious adverse events and febrile neutropenia. Similarly, Decitabine–Cedazuridine exposure was individualized over time, supporting a treatment paradigm that emphasizes intensive disease control during induction followed by tailored maintenance of remission.

Clinical Implications for Practice

The ASCERTAIN-V results reinforce the growing recognition that effective AML treatment can potentially be delivered outside traditional infusion-based settings.

For clinicians, the findings highlight several practical considerations:

  • Early bone marrow evaluation may help identify patients who can benefit from abbreviated Venetoclax exposure.
  • Dose modifications should be incorporated proactively to mitigate prolonged cytopenias.
  • Growth factor support should be considered in patients with severe or persistent neutropenia.
  • Venetoclax dosing must be adjusted appropriately when azole antifungal prophylaxis is required.
  • Close monitoring remains essential despite the convenience of oral administration.

The trial also underscores the importance of adherence monitoring, as treatment success with oral regimens depends on consistent medication use. Investigators reported high adherence rates through the use of patient diaries and pill counts.

A Potential Alternative to Infusion-Based HMA-Venetoclax Therapy

Cross-trial comparisons should be interpreted cautiously, particularly given the single-arm design of ASCERTAIN-V. Nevertheless, the observed remission rates, overall survival outcomes, and safety profile appear broadly comparable to those reported historically with Azacitidine-Venetoclax and IV Decitabine-Venetoclax combinations.

Given that oral Decitabine–Cedazuridine achieves systemic exposure equivalent to intravenous Decitabine, the study supports the concept that an all-oral HMA-Venetoclax regimen can deliver clinically meaningful outcomes without introducing new safety concerns.

Beyond efficacy, the potential reduction in travel, clinic visits, caregiver burden, and treatment-associated disruption may represent a significant advantage for older patients who often face mobility limitations and competing medical challenges.

Looking Ahead

The ASCERTAIN-V trial marks an important step toward more patient-centered AML care. By demonstrating meaningful remission rates, durable responses, and survival outcomes with a fully oral treatment strategy, the study provides evidence that convenience and clinical efficacy do not necessarily need to be mutually exclusive.

While randomized comparative studies and formal Quality-of-Life assessments would further strengthen the evidence base, these findings suggest that oral Decitabine–Cedazuridine plus Venetoclax may emerge as a valuable frontline option for patients with newly diagnosed AML who are not candidates for intensive chemotherapy.

As the AML treatment landscape continues to evolve, the ability to deliver effective therapy through a fully oral regimen has the potential to reshape care delivery and improve the treatment experience for a vulnerable patient population.

All-Oral Treatment of Newly Diagnosed Acute Myeloid Leukemia. Roboz GJ,  Zeidan AM, Mannis GN, et al. N Engl J Med 2026;394:2107-2116.

FDA Approves KOMZIFTI® for Relapsed or Refractory Acute Myeloid Leukemia with a NPM1 mutation

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SUMMARY: The FDA on November 13, 2025, approved Ziftomenib (KOMZIFTI®), a menin inhibitor, for adults with relapsed or refractory Acute Myeloid Leukemia (AML) with a susceptible Nucleophosmin 1 (NPM1) mutation who have no satisfactory alternative treatment options.

The American Cancer Society estimates that in 2025, 22,010 new cases of Acute Myeloid Leukemia (AML) will be diagnosed in the United States and 11,090 patients will die of the disease. AML is one of the most common types of leukemia in adults and can be considered as a group of molecularly heterogeneous diseases with different clinical behavior and outcomes. With the understanding of molecular pathology of AML, personalized and targeted therapies are becoming an important part of the AML treatment armamentarium.

NPM1 mutations present in up to 30% of newly diagnosed adult AML define a unique disease subset recognized by both the WHO and the International Consensus Classification. Although NPM1-mutated AML often responds well to initial intensive therapy, outcomes deteriorate sharply after relapse or refractory disease. Patients in this setting frequently face limited therapeutic options and dismal response rates with conventional salvage strategies.

Mounting evidence shows that NPM1-mutated and KMT2A-rearranged (KMT2A-r) leukemias rely on an aberrant transcriptional program maintained through the interaction between menin and KMT2A. This dependency includes pathologic overexpression of HOX and MEIS1, which reinforce leukemic self-renewal and block differentiation. Inhibiting the menin–KMT2A complex has therefore emerged as a compelling therapeutic strategy capable of reversing leukemic transcriptional programs.

Ziftomenib, a potent and selective oral menin inhibitor, disrupts this interaction and restores myeloid differentiation in preclinical models. The registrational Phase II portion of the KOMET-001 study provides the most definitive evidence to date of its clinical potential in relapsed/refractory NPM1-mutated AML, and was used for the primary efficacy analysis and formed the basis for the FDA approval.

Mechanistic Rationale for Menin Inhibition

Menin serves as a scaffold protein essential for recruitment of the KMT2A/MLL methyltransferase complex to chromatin. This interaction drives leukemogenic transcriptional circuits in both KMT2A-r and NPM1-mutated AML, promoting expression of HOXA9, MEIS1, PBX3, and downstream effectors such as FLT3 and BCL2.

Key mechanistic insights include:

  • Menin–KMT2A blockade releases mutant NPM1 from chromatin, reducing HOX/MEIS1 signaling and triggering differentiation.
  • Ziftomenib promotes terminal maturation of AML blasts, rather than direct cytotoxicity, consistent with its differentiation-based mechanism.
  • NPM1 cytoplasmic mislocalization, a hallmark of the mutation, creates vulnerabilities that can be exploited through menin inhibition and related targeted approaches.

This biology underpins the therapeutic activity observed in KOMET-001 and supports the broader pursuit of menin inhibition across multiple AML subtypes.

KOMET-001 Trial Overview

KOMET-001 is a global, multicenter Phase I/II study evaluating single-agent Ziftomenib in adults with relapsed/refractory NPM1-mutated or KMT2A-rearranged AML. The Phase II portion which serves as the registrational dataset, focused on patients with relapsed/refractory NPM1-mutated disease treated at the recommended monotherapy dose of Ziftomenib 600 mg once daily.

Among the 92 patients with relapsed/refractory NPM1-mutated AML included in Phase II:

  • Median age: 69 years (range 33–84); 64% were ≥65 years
  • Median prior therapies: 2 lines (range 1–7)
  • Prior venetoclax exposure: 59%
  • Prior allogeneic transplantation: 24%
  • Common co-mutations: FLT3 (56%), IDH1/2 (33%)
  • ECOG 0–1: 83%

Efficacy was established based on the rate of Complete Remission (CR) plus CR with partial hematological recovery (CRh), the duration of CR plus CRh, and the rate of conversion from transfusion dependence to transfusion independence. The median follow-up was 4.2 months.

This heavily pretreated population reflects real-world patients with few remaining therapeutic options and particularly poor expected outcomes.

Efficacy Findings

Ziftomenib met its Primary endpoint with a CR/CRh rate of 22% (95% CI 14–32; P=0.0058), exceeding the historical 12% benchmark for this setting.

Key efficacy results:

  • CR/CRh rate: 22% (14% CR; 8% CRh)
  • Composite CR rate: 26%
  • Overall Response Rate (ORR): 33%
  • Median time to first response: 1.9 months
  • Median duration of response: 4.6 months
  • MRD negativity: 61% of evaluable CR/CRh responders
  • Median Overall Survival (OS): 6.6 months
  • Median OS among responders: 18.4 months

Two responders were successfully bridged to allogeneic stem cell transplantation and resumed Ziftomenib maintenance afterwards.

Efficacy was maintained across clinically relevant subgroups:

  • Age <65 vs ≥65: 21% vs 22% CR/CRh
  • Prior venetoclax exposure: 22% CR/CRh
  • Prior HSCT: 23% CR/CRh
  • FLT3 co-mutations: 13% (ITD), 33% (TKD)
  • IDH1/2 co-mutations: 50% and 31%, respectively

The activity in Venetoclax-exposed patients is particularly noteworthy given real-world salvage CR rates as low as 4% in this population.

Transfusion Independence

  • RBC independence conversion: 23%
  • Platelet independence conversion: 15%
  • Overall transfusion independence conversion: 20%

These improvements reflect meaningful clinical benefit and enhanced quality of life.

Safety and Tolerability

Ziftomenib demonstrated a favorable safety profile with low rates of treatment-related discontinuation (3%). Most adverse events were consistent with underlying AML or expected from differentiation-based therapies. Common Grade ≥3 TEAEs included febrile neutropenia (26%), anemia (20%) and thrombocytopenia (20%). Differentiation Syndrome occurred in 25% (15% grade 3; none grade 4–5) managed effectively using protocol-defined measures including cytoreduction and steroid prophylaxis, reinforcing the necessity of early recognition and continued therapy through differentiation-associated changes. Overall, ziftomenib showed no clear intrinsic myelosuppression, minimal cardiac toxicity, and a manageable safety profile appropriate for a predominantly older patient population.

Clinical Implications

The KOMET-001 data establish Ziftomenib as a meaningful therapeutic advance for patients with relapsed/refractory NPM1-mutated AML, an area historically characterized by low response rates and short survival. The durability of responses, high rate of MRD clearance, and consistent efficacy across age groups, co-mutational backgrounds, and prior therapies position Ziftomenib as a valuable monotherapy option and a potential bridge to curative transplantation. These results also strengthen the biological rationale for integrating menin inhibitors earlier in the treatment course. Ongoing frontline trials including the global Phase III KOMET-017 study will clarify the role of Ziftomenib-based combinations in newly diagnosed fit and unfit patients with NPM1-mutated or KMT2A-rearranged AML.

Conclusion

Ziftomenib, a first-in-class oral menin inhibitor, demonstrated clinically meaningful activity and durable responses in heavily pretreated relapsed/refractory NPM1-mutated AML, meeting its registrational Phase II endpoint. With a manageable safety profile including low myelosuppression, minimal QTc effects, and predictable differentiation syndrome, Ziftomenib represents an important new targeted therapy for a genetically defined AML subset.

The KOMET-001 results mark a significant step forward in addressing an area of profound unmet need, and they lay the foundation for expanding menin inhibition into earlier lines of therapy with the goal of transforming long-term outcomes for patients with NPM1-mutated AML.

Ziftomenib in Relapsed or Refractory NPM1-Mutated AML. Wang ES, Montesinos P, Foran J, et al. J Clin Oncol. 2025;43:3381-3390

FDA Approves REVUFORJ® for Acute Leukemia with KMT2A Translocation

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SUMMARY: The FDA on November 15, 2024, approved Revumenib (REVUFORJ®), a menin inhibitor, for Relapsed or Refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients 1 year and older. The American Cancer Society estimates that in 2024, 20,800 new cases of Acute Myeloid Leukemia (AML) will be diagnosed in the United States and 11,220 patients will die of the disease. AML is one of the most common types of leukemia in adults and can be considered as a group of molecularly heterogeneous diseases with different clinical behavior and outcomes. A significant percentage of patients with newly diagnosed AML are not candidates for intensive chemotherapy or have disease that is refractory to standard chemotherapy. Even with the best available therapies, the 5-year Overall Survival in patients 65 years of age or older is less than 5%. Cytogenetic analysis has been part of routine evaluation when caring for patients with AML. By predicting resistance to therapy, tumor cytogenetics will stratify patients based on risk, and help manage them accordingly. Even though cytotoxic chemotherapy may lead to long term remission and cure in a minority of patients with favorable cytogenetics, patients with high-risk features such as unfavorable cytogenetics, molecular abnormalities, prior myelodysplasia, and advanced age, have poor outcomes with conventional chemotherapy alone. More importantly, with the understanding of molecular pathology of AML, personalized and targeted therapies are becoming an important part of the AML treatment armamentarium. Over 50% of AML cases lack targetable mutations, relying instead on toxic chemotherapy.

Rearrangements of KMT2A gene previously known as MLL are found in 80% of infant Acute Lymphoblastic Leukemia (ALL) and in 5-15% of acute leukemia cases in children and adults, including myeloid, lymphoid, or mixed phenotypes. NPM1 mutations are the most common genetic alteration in adult Acute Myeloid Leukemia (AML), occurring in up to 30% of cases. Acute leukemias with KMT2A rearrangements have a poor prognosis, with a 5-year overall survival rate of less than 25%. There are no targeted therapies currently approved specifically for acute leukemia with KMT2A gene rearrangements or NPM1 mutations. Both KMT2A gene rearrangements and NPM1 mutations cause blood cells to regress to a stem-cell-like state, leading to the formation of leukemia cells. For leukemias driven by KMT2A gene rearrangements and NPM1 mutations, menin is a critical oncogenic cofactor. Menin interacts with the protein MLL1 (produced by KMT2A), forming a menin–MLL1 complex. This complex binds to chromatin and activates aberrant gene pathways, specifically HOX genes and their cofactor MEIS1, critical for leukemia development.

Revumenib is a potent, oral, small molecule menin inhibitor. It blocks the menin–MLL1 interaction, preventing the formation of the menin–MLL1 complex. By disrupting this complex, Revumenib stops the aberrant activation of HOX and MEIS1 gene expression and allows leukemia cells to either die or differentiate back into normal blood cells. Unlike other targeted therapies that block dysfunctional proteins, Revumenib prevents aberrant gene expression at its source. Its ability to target a common mechanism in AML makes it broadly applicable. Preclinical Studies demonstrated that menin inhibition reverses leukemia progression by downregulating HOX and MEIS1 transcription disrupting oncogenic complexes formed by either option for patients with KMT2A gene arrangements or NPM1-mutated AML. Revumenib showed dramatic antileukemic activity, making this agent promising.

AUGMENT-101 is a single-arm cohort of an open-label, multicenter trial which included 104 adult and pediatric patients (at least 30 days old) with Relapsed or Refractory (R/R) acute leukemia with a KMT2A translocation. Eligible patients had a corrected QT interval of less than 450 milliseconds and those with an11q23 partial tandem duplication were excluded. Revumenib was administered at a dose that was approximately equivalent to 160 mg in adults orally twice daily. Treatment was continued until progressive disease, unacceptable toxicity, failure to achieve a morphological leukemia-free state by 4 cycles of treatment, or Hematopoietic Stem Cell Transplantation (HSCT). The median patient age was 37 years, 83% of patients had AML, 15% had Acute Lymphoblastic Leukemia, and 2% had mixed phenotype acute leukemia. Approximately 59% had relapsed/refractory disease, 21% had primary refractory disease, 20% of patients had untreated relapsed disease and 44% of patients underwent prior HSCT. The main efficacy outcome measures were Complete Remission (CR) plus CR with partial hematologic recovery (CRh), the duration of CR plus CRh, and conversion from transfusion dependence to independence.

The CR plus CRh rate was 21.2%, and the median CR plus CRh duration was 6.4 months. Of the 22 patients achieving CR or CRh, the median time to CR or CRh was 1.9 months. Among the 83 patients dependent on RBC and/or platelet transfusions at baseline, 14% became independent of RBC and platelet transfusions during any 56-day post-baseline period. Of the 21 patients independent of both RBC and platelet transfusions at baseline, 48% remained transfusion independent during any 56-day post-baseline period. The most common adverse reactions noted in this study were hemorrhage, nausea, increased phosphate, musculoskeletal pain, neutropenia, infection, elevated liver enzymes, differentiation syndrome, QT prolongation and fatigue.

It was concluded that Revumenib is the first menin inhibitor and its efficacy represents a substantial improvement over previously available therapies, and represents a major breakthrough for patients with Relapsed or Refractory acute leukemia with a KMT2A translocation.

https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-revumenib-relapsed-or-refractory-acute-leukemia-kmt2a-translocation

Late Breaking Abstract- ASH 2023: ERG is a New Predisposition Gene for Bone Marrow Failure and Hematological Malignancy

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SUMMARY: ERG (ETS-Related Gene) is a known oncogene located on chromosome 21, and is a member of the ETS (erythroblast transformation-specific) family of transcription factors. The ERG gene encodes for a protein also called ERG that functions as a transcriptional regulator, and regulates differentiation of early hematopoietic cells.

ERG has been linked to Down syndrome- associated Acute Megakaryocytic Leukemia. ERG typically via gene-fusions can lead to dysregulated ERG overexpression in hematologic malignancies and solid tumors. ERG can fuse with TMPRSS2 protein to form an oncogenic fusion gene that is commonly found in Hormone-Refractory Prostate Cancer, suggesting that ERG overexpression may contribute to development of androgen-independence in prostate cancer through disruption of androgen receptor signaling. EWS1-ERG fusion has been noted in 10% of Ewing’s Sarcoma cases. ERG is also involved in oncogenesis by generating fusion genes with FUS/TLS in Acute Myeloid Leukemia.

The researchers in this study identified a germline ERG variant associated with bone marrow failure and hematological malignancies. The study originated from a family case involving thrombocytopenia and neutropenia, where the mother developed Acute Myeloid Leukemia (AML) and MyeloDysplastic Syndrome (MDS). ERG, a known oncogene, was discovered as a predisposition gene for bone marrow failure and hematological malignancy. A germline ERG ETS domain variant (p.Y373C) was identified, segregating with thrombocytopenia in a family, leading to AML and therapy-related MDS. Copy neutral Loss of Heterozygosity of chromosome 21q, including the ERG locus, was observed in affected individuals. Validation of their findings involved functional assays, demonstrating Loss of Function variants in ERG, affecting DNA binding and nuclear localization. Experiments using a fetal liver assay confirmed the role of ERG in cytokine-independent growth and leukemia development.

Through global collaborations, 15 heterozygous variants in the ERG gene were identified, including 13 missense and 2 truncating variants in 17 individuals with cytopenia, hematological malignancy or lymphedema. Of these 15 variants, 12 have been confirmed germline. Onset of hematological symptoms ranged from birth to 38 years for truncating and constrained ETS domain variants. Functional studies revealed that most ETS domain missense variants displayed Loss-of-Function (LOF) characteristics affecting transcriptional transactivation, DNA binding, and/or nuclear localization.

This ERG syndrome parallels GATA2 deficiency syndrome (hematological malignancy with lymphedema) and RUNX1 Familial Platelet disorder-myeloid malignancy (thrombocytopenia and hematological malignancy). ERG, like the well-known disease genes GATA2, and RUNX1 is a member of the transcription factor heptad involved in hematopoietic stem cell maintenance and differentiation.

The researchers concluded that germline ERG variants predispose to diverse cytopenia, bone marrow failure and hematological malignancies in both children and adults and ERG adds to a growing list of genes whose unregulated expression contributes to hematological malignancy and other cancers. Identification of germline ERG variants has direct clinical implications for patient and family management including diagnosis, counseling, surveillance, and treatment strategies, such as bone marrow transplant and targeted therapies. Potential clinical implications include ERG screening in germline panels for bone marrow failures and hematological malignancies. Additionally there is a need for further longitudinal studies to understand the natural history of ERG-related syndromes.

ERG is a New Predisposition Gene for Bone Marrow Failure and Hematological Malignancy. Scott HS, Zerella J, Homan C, et al. ASH Annual Meeting & Exposition 2023. LBA-6.

VANFLYTA® (Quizartinib)

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The FDA on July 20, 2023, approved VANFLYTA® (Quizartinib) 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. VANFLYTA® is a product of Daiichi Sankyo, Inc.