Advocacy intelligence hub — real-time data for patient organizations
In combination with azacitidine or as monotherapy for the treatment of newly diagnosed acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy
XOSPATA: FDA approved
XOSPATA is indicated for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FMS-like tyrosine kinase 3 (FLT3) mutation as detected by an FDA-approved test.
DAURISMO: FDA approved
DAURISMO is indicated, in combination with low-dose cytarabine, for the treatment of newly-diagnosed acute myeloid leukemia (AML) in adult patients who are >75 years old or who have comorbidities that preclude use of intensive induction chemotherapy.
Roswell Park Cancer Institute — PHASE1
Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
FINANCIAL LANDSCAPE SUMMARY
Total programs
Open now
Patient Assistance Programs2
DAURISMO
Pfizer, Inc.
Idamycin
Adria Laboratories, Inc.
Tibsovo
(IVOSIDENIB)Orphan drugServier Pharmaceutical LLC
Isocitrate Dehydrogenase 1 Inhibitor [EPC]
12.1 Mechanism of Action Ivosidenib is a small molecule inhibitor that targets the mutant isocitrate dehydrogenase 1 (IDH1) enzyme. In patients with A...
XOSPATA
(gilteritinib)Orphan drugAstellas Pharma US, Inc.
12.1 Mechanism of Action Gilteritinib is a small molecule that inhibits multiple receptor tyrosine kinases, including FMS-like tyrosine kinase 3 (FLT3...
DAURISMO
(Glasdegib)Orphan drugPfizer, Inc.
Hedgehog Pathway Inhibitor [EPC]
12.1 Mechanism of Action Glasdegib is an inhibitor of the Hedgehog pathway. Glasdegib binds to and inhibits Smoothened, a transmembrane protein involv...
Idamycin
(Idarubicin HCl for injection)Orphan drugstandardAdria Laboratories, Inc.
12.1 Mechanism of Action Idarubicin hydrochloride has antimitotic and cytotoxic activity through forming complexes with the DNA, inhibiting nucleic ac...
[Acute megakaryoblastic leukemia with CBFA2T3::GLIS2 fusion gene: 3 cases report and literature review].
This study retrospectively analyzed the clinical manifestations, genetic characteristics, treatment courses, and prognoses of patients with pediatric acute megakaryoblastic leukemia (AMKL) with CBFA2T...
Identification of an Elusive CBFA2T3::GLIS2 Fusion Variant in Acute Megakaryoblastic Leukemia by Whole Genome Sequencing.
Advancements in genomic profiling have significantly improved the classification and treatment strategies for acute myeloid leukemia (AML). However, widely utilized molecular diagnostic techniques, in...
BH3 mimetic therapies for CBFA2T3::GLIS2 pediatric acute megakaryoblastic leukemia.
Pediatric acute megakaryoblastic leukemia (AMKL) associated with the CBFA2T3::GLIS2 (CG2) gene fusion is distinctive by virtue of its aggressiveness and ability to transform normal cells in a single e...
Polycomb repressive complex 2 insufficiency underlies myeloid leukemia in Down syndrome.
Children with Down syndrome (DS) have an elevated risk of developing myeloid leukemia in DS (ML-DS). In addition to mutations in GATA1, which generate the truncated isoform GATA1-short (GATA1s), ML-DS...
Acute megakaryoblastic leukemia with myeloid sarcoma among a pediatric patient harbouring RBM15::MRTFA: a case report and literature review.
Acute megakaryoblastic leukemia with myeloid sarcoma among a pediatric patient harbouring RBM15::MRTFA: a case report and literature review.
Acute megakaryoblastic leukemia with RBM15::MKL1 fusion presenting as neonatal acute liver failure: rescued by living-donor liver transplantation.
Acute liver failure in the neonatal period is commonly caused by infections, metabolic disorders, immune disorders, or neonatal hepatitis-like diseases of unknown etiology; however, malignant diseases...
Soheil Meshinchi, MD, MD,PhD
Fred Hutchinson Cancer Center
📍 SEATTLE, WA
B. Smith, M.S.W., L.C.S.W
Johns Hopkins University
📍 OKLAHOMA CITY, OK
Kyoo H. Lee, MD
Asan Medical Center
Colleen Delaney, MD
Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
📍 OLD GREENWICH, CT
Sucha Nand, MD
SWOG Cancer Research Network
📍 MAYWOOD, IL
Jeffrey Lancet, MD
Moffitt Cancer Center
📍 TAMPA, FL