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Gilteritinib

Generic Name
Gilteritinib
Brand Names
Xospata
Drug Type
Small Molecule
Chemical Formula
C29H44N8O3
CAS Number
1254053-43-4
Unique Ingredient Identifier
66D92MGC8M
Background

Gilteritinib, also known as ASP2215, is a small molecule part of the FLT3 tyrosine kinase inhibitors that presented a greater selectivity and potency when compared with other agents from this group. It is a pyrazinecarboxamide derivative that showed high selectivity to FLT3 preventing the c-Kit -driven myelosuppression observed in other therapies. Gilteritinib was developed by Astellas Pharma and FDA approved on November 28, 2018. This drug was approved after being designed as an orphan drug with a fast track and priority review status.

Indication

Gilteritinib is indicated for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia with an FLT3 mutation detected by an FDA-approved test. This indication was expanded for a companion diagnostic to include use with gilteritinib such as the LeukoStrat CDx FLT3 Mutation Assay.

Acute myeloid leukemia is cancer that impacts the blood and bone marrow with a rapid progression. This condition produces low numbers of normal blood cells and the requirement of continuous need for transfusions.

Associated Conditions
Relapsed or Refractory Acute Myeloid Leukemia With FLT3 Activating Mutations

Astellas to Present New Long-Term Survival Data for Cancer Therapies at ASCO 2025

• Astellas will showcase 16 abstracts at the 2025 ASCO Annual Meeting, including two oral presentations highlighting long-term survival data for its oncology portfolio. • New post-hoc analyses will feature five-year overall survival data for XTANDI (enzalutamide) in metastatic hormone-sensitive prostate cancer and exploratory analyses for PADCEV (enfortumab vedotin) in urothelial cancer. • The presentations underscore Astellas' commitment to delivering meaningful clinical outcomes for patients with advanced prostate and bladder cancers through innovative treatment approaches.

Invivoscribe's LeukoStrat CDx FLT3 Mutation Assay Receives Regulatory Approval in UK and Switzerland

• The LeukoStrat CDx FLT3 Mutation Assay has been officially registered with regulatory agencies in the United Kingdom and Switzerland, expanding access to critical diagnostics for acute myeloid leukemia patients. • The PCR-based diagnostic test enables same-day detection of FLT3 mutations, which are associated with poor prognosis in AML, helping clinicians make timely and informed treatment decisions. • The assay serves as a companion diagnostic for targeted therapies including XOSPATA (gilteritinib fumarate) and VANFLYTA (quizartinib hydrochloride) in regions where these treatments are available.

Emavusertib Shows Promise in FLT3-Mutated AML Patients Resistant to Prior Therapies

• Phase 1/2 TakeAim Leukemia trial demonstrates significant efficacy of emavusertib in relapsed/refractory AML patients, with 6 complete remissions observed among 19 evaluable FLT3-mutated patients. • The IRAK4 inhibitor showed effectiveness in patients previously treated with FLT3 inhibitors like midostaurin and gilteritinib, suggesting potential to overcome existing resistance mechanisms. • Clinical responses were also observed in patients with spliceosome factor mutations, including those heavily pretreated with hypomethylating agents plus venetoclax.

Oryzon's Iadademstat Enters Phase I Trial for Myelodysplastic Syndrome

• Oryzon Genomics has announced the dosing of the first patient in a Phase I trial of iadademstat for myelodysplastic syndrome (MDS). • The trial, led by the Medical College of Wisconsin, will assess the safety, tolerability, and optimal dose of iadademstat with azacitidine. • Iadademstat, a selective LSD1 inhibitor, aims to address the differentiation block in hematopoietic progenitor cells characteristic of MDS. • The study hopes to improve outcomes for MDS patients, where current treatments have limited efficacy, especially in higher-risk cases.

Daiichi Sankyo's Vanflyta Enters Phase 3 Trial for FLT3-ITD Negative AML

• Daiichi Sankyo initiates the QuANTUM-Wild phase 3 trial to evaluate Vanflyta (quizartinib) in FLT3-ITD negative acute myeloid leukemia (AML). • The trial will assess Vanflyta combined with chemotherapy and as maintenance monotherapy for newly diagnosed AML patients. • QuANTUM-Wild is a randomized, double-blind, placebo-controlled study aiming to enroll approximately 700 patients globally. • The primary endpoint is overall survival, with secondary endpoints including event-free survival and complete remission rate.

Kronos Bio Halts Istisociclib Development, Considers Strategic Alternatives

• Kronos Bio discontinued istisociclib (KB-0742) development after safety concerns arose in a Phase I/II trial for platinum-resistant high-grade serous ovarian cancer. • The trial revealed neurological adverse events in five of seven patients, leading to treatment discontinuation or dosage reduction in some participants. • Facing financial pressures, Kronos Bio is exploring strategic alternatives, including acquisition or merger, to maximize stockholder value and preserve cash. • The company is also seeking partnerships for preclinical assets KB-9558 and KB-7898, while focusing on IND application for multiple myeloma and autoimmune disorders.

LeukoStrat® CDx FLT3 Mutation Assay Receives EU Approval for AML Treatment Selection

• Invivoscribe's LeukoStrat® CDx FLT3 Mutation Assay has been approved by BSI (Netherlands) and the EMA. • The assay aids in identifying acute myeloid leukemia (AML) patients eligible for VANFLYTA® (quizartinib) treatment in the EU. • The LeukoStrat® CDx FLT3 Mutation Assay detects FLT3-ITD and TKD mutations in AML patients. • This approval provides oncologists with a tool to optimize treatment strategies for AML patients with FLT3 mutations.
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