| Anagrelide | US FDA | Myeloproliferative Neoplasm | AGRYLIN is indicated for the treatment of patients with thrombocythemia, secondary to myeloproliferative neoplasms, to reduce the elevated platelet count and the risk of thrombosis and to ameliorate associated symptoms including thrombo-hemorrhagic events. AGRYLIN is a platelet reducing agent indicated for the treatment of thrombocythemia, secondary to myeloproliferative neoplasms, to reduce the elevated platelet count and the risk of thrombosis and to ameliorate associated symptoms including thrombo-hemorrhagic events. ( 1 ) | approved | Mar 14, 1997 | openfda |
| Arsenic Trioxide | US FDA | Acute Promyelocytic Leukemia | In combination with tretinoin for treatment of adults with newly-diagnosed low-risk acute promyelocytic leukemia (APL) whose APL is characterized by the presence of the t(15;17) translocation or PML/RAR-alpha gene expression. | approved | Sep 25, 2000 | openfda |
| Azacitidine | US FDA | Acute Myeloid Leukemia | ONUREG is indicated for continued treatment of adult patients with acute myeloid leukemia who achieved first complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following intensive induction chemotherapy and are not able to complete intensive curative therapy. ONUREG is a nucleoside metabolic inhibitor indicated for continued treatment of adult patients with acute myeloid leukemia who achieved first complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following intensive induction chemotherapy and are not able to complete intensive curative therapy ( 1 ). | approved | Sep 1, 2020 | openfda |
| Azacitidine | US FDA | Juvenile Myelomonocytic Leukemia | Pediatric patients aged 1 month and older with newly diagnosed Juvenile Myelomonocytic Leukemia (JMML). | approved | May 19, 2004 | openfda |
| Bosutinib | US FDA | Chronic Myeloid Leukemia, Philadelphia Chromosome Negative, BCR-ABL1 Positive | adult and pediatric patients 1 year of age and older with chronic phase Ph+ chronic myelogenous leukemia (CML), newly-diagnosed or resistant or intolerant to prior therapy. ( 1 ) | approved | Sep 26, 2023 | openfda |
| Bosutinib | US FDA | Chronic Myeloid Leukemia, Philadelphia Chromosome Negative, BCR-ABL1 Positive | adult and pediatric patients 1 year of age and older with chronic phase Ph+ chronic myelogenous leukemia (CML), newly-diagnosed or resistant or intolerant to prior therapy. ( 1 ) | approved | Sep 4, 2012 | openfda |
| Busulfan | US FDA | Myeloid Leukemia | MYLERAN (busulfan) is indicated for the palliative treatment of chronic myelogenous (myeloid, myelocytic, granulocytic) leukemia. | approved | Jun 26, 1954 | openfda |
| Dabrafenib | US FDA | Tumor-agnostic | the treatment of adult and pediatric patients 1 year of age and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment and have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on overall response rate (ORR) and duration of response (DoR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). • the treatment of pediatric patients 1 year of age and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy. • TAFINLAR is not indicated for treatment of patients with wild-type BRAF solid tumors. ( 5.2 )accelerated | approved | Mar 16, 2023 | openfda |
| Dabrafenib | US FDA | Tumor-agnostic | the treatment of adult and pediatric patients 1 year of age and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment and have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on overall response rate (ORR) and duration of response (DoR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). • the treatment of pediatric patients 1 year of age and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy. • TAFINLAR is not indicated for treatment of patients with wild-type BRAF solid tumors. ( 5.2 )accelerated | approved | May 29, 2013 | openfda |
| Dostarlimab | US FDA | Tumor-agnostic | as a single agent for the treatment of adult patients with mismatch repair deficient (dMMR) recurrent or advanced EC, as determined by an FDA-approved test, that has progressed on or following prior treatment with a platinum-containing regimen in any setting and are not candidates for curative surgery or radiation. • Mismatch Repair Deficient Recurrent or Advanced Solid Tumors • as a single agent for the treatment of adult patients with dMMR recurrent or advanced solid tumors, as determined by an FDA-approved test, that have progressed on or following prior treatment and who have no satisfactory alternative treatment options. 1 • 1 This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).accelerated | approved | Apr 22, 2021 | openfda |
| Enasidenib | US FDA | Acute Myeloid Leukemia | IDHIFA is an isocitrate dehydrogenase-2 inhibitor indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA-approved test | approved | Aug 1, 2017 | openfda |
| Entrectinib | US FDA | Tumor-agnostic | Adult and pediatric patients older than 1 month of age with solid tumors that: have a neurotrophic tyrosine receptor kinase ( NTRK) gene fusion, as detected by an FDA-approved test without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory alternative therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.accelerated | approved | Oct 20, 2023 | openfda |
| Entrectinib | US FDA | Tumor-agnostic | Adult and pediatric patients older than 1 month of age with solid tumors that: have a neurotrophic tyrosine receptor kinase ( NTRK) gene fusion, as detected by an FDA-approved test without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory alternative therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.accelerated | approved | Aug 15, 2019 | openfda |
| Filgrastim | US FDA | Acute Myeloid Leukemia | Reduce the time to neutrophil recovery and the duration of fever, following induction or consolidation chemotherapy treatment of patients with acute myeloid leukemia (AML). | approved | Feb 25, 2022 | openfda |
| Filgrastim | US FDA | Acute Myeloid Leukemia | Reduce the time to neutrophil recovery and the duration of fever, following induction or consolidation chemotherapy treatment of patients with acute myeloid leukemia (AML) | approved | Feb 20, 1991 | openfda |
| Gemtuzumab Ozogamicin | US FDA | Acute Myeloid Leukemia | treatment of newly-diagnosed CD33-positive acute myeloid leukemia (AML) in adults and pediatric patients 1 month and older | approved | Sep 1, 2017 | openfda |
| Gemtuzumab Ozogamicin | US FDA | Acute Myeloid Leukemia | treatment of newly-diagnosed CD33-positive acute myeloid leukemia (AML) in adults and pediatric patients 1 month and older | approved | Sep 1, 2017 | openfda |
| Gilteritinib | US FDA | Acute Myeloid Leukemia | XOSPATA is a kinase inhibitor indicated for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation as detected by an FDA-approved test. | approved | Nov 28, 2018 | openfda |
| Glasdegib | US FDA | Acute Myeloid Leukemia | 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. DAURISMO is a hedgehog pathway inhibitor 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. ( 1 ) | approved | Nov 21, 2018 | openfda |
| Idarubicin | US FDA | Acute Myeloid Leukemia | IDAMYCIN PFS is indicated for the treatment of adult patients with acute myeloid leukemia (AML) as a component of a combination chemotherapy regimen. IDAMYCIN PFS is an anthracycline topoisomerase inhibitor indicated for the treatment of adult patients with acute myeloid leukemia (AML) as a component of a combination chemotherapy regimen. (1) | approved | Feb 17, 1997 | openfda |
| Imatinib | US FDA | Chronic Eosinophilic Leukemia, Not Otherwise Specified | Adult patients with hypereosinophilic syndrome (HES) and/or chronic eosinophilic leukemia (CEL) who have the FIP1L1-PDGFRα fusion kinase (mutational analysis or fluorescence in situ hybridization [FISH] demonstration of CHIC2 allele deletion) and for patients with HES and/or CEL who are FIP1L1-PDGFRα fusion kinase negative or unknown. | approved | Nov 22, 2024 | openfda |
| Imatinib | US FDA | Myelodysplastic/Myeloproliferative Neoplasm | Adult patients with myelodysplastic/myeloproliferative diseases (MDS/MPD) associated with platelet-derived growth factor receptor (PDGFR) gene re-arrangements. | approved | Nov 22, 2024 | openfda |
| Imatinib | US FDA | Myelodysplastic/Myeloproliferative Neoplasm | Adult patients with myelodysplastic/myeloproliferative diseases (MDS/MPD) associated with platelet-derived growth factor receptor (PDGFR) gene re-arrangements. | approved | Apr 18, 2003 | openfda |
| Imatinib | US FDA | Chronic Eosinophilic Leukemia, Not Otherwise Specified | Adult patients with hypereosinophilic syndrome (HES) and/or chronic eosinophilic leukemia (CEL) who have the FIP1L1-PDGFRα fusion kinase (mutational analysis or fluorescence in situ hybridization [FISH] demonstration of CHIC2 allele deletion) and for patients with HES and/or CEL who are FIP1L1-PDGFRα fusion kinase negative or unknown. | approved | Apr 18, 2003 | openfda |
| Imatinib Mesylate | US FDA | Myelodysplastic/Myeloproliferative Neoplasm | Adult patients with myelodysplastic/myeloproliferative diseases (MDS/MPD) associated with platelet-derived growth factor receptor (PDGFR) gene re-arrangements. | approved | Apr 18, 2003 | openfda |
| Imatinib Mesylate | US FDA | Chronic Eosinophilic Leukemia, Not Otherwise Specified | Adult patients with hypereosinophilic syndrome (HES) and/or chronic eosinophilic leukemia (CEL) who have the FIP1L1-PDGFRα fusion kinase (mutational analysis or fluorescence in situ hybridization [FISH] demonstration of CHIC2 allele deletion) and for patients with HES and/or CEL who are FIP1L1-PDGFRα fusion kinase negative or unknown. | approved | Apr 18, 2003 | openfda |
| Ivosidenib | US FDA | Myelodysplastic Syndrome | . Relapsed or refractory Myelodysplastic Syndromes (MDS) For the treatment of adult patients with relapsed or refractory myelodysplastic syndromes | approved | Jul 20, 2018 | openfda |
| Ivosidenib | US FDA | Acute Myeloid Leukemia | Newly Diagnosed Acute Myeloid Leukemia (AML) In combination with azacitidine or as monotherapy for the treatment of newly diagnosed AML in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy | approved | Jul 20, 2018 | openfda |
| Larotrectinib | US FDA | Tumor-agnostic | VITRAKVI is indicated for the treatment of adult and pediatric patients with solid tumors that: have a neurotrophic receptor tyrosine kinase ( NTRK ) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have no satisfactory alternative treatments or that have progressed following treatment. Select patients for therapy based on an FDA-approved test [see Dosage and Administration (2.1) ]. VITRAKVI is a kinase inhibitor indicated for the treatment of adult and pediatric patients with solid tumors that: have a neurotrophic receptor tyrosine kinase ( NTRK ) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have no satisfactory alternative treatments or that have progressed following treatment. Select patients for therapy based on an FDA-approved test. ( 1 , 2.1 ) | approved | Nov 26, 2018 | openfda |
| Larotrectinib | US FDA | Tumor-agnostic | VITRAKVI is indicated for the treatment of adult and pediatric patients with solid tumors that: have a neurotrophic receptor tyrosine kinase ( NTRK ) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have no satisfactory alternative treatments or that have progressed following treatment. Select patients for therapy based on an FDA-approved test [see Dosage and Administration (2.1) ]. VITRAKVI is a kinase inhibitor indicated for the treatment of adult and pediatric patients with solid tumors that: have a neurotrophic receptor tyrosine kinase ( NTRK ) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have no satisfactory alternative treatments or that have progressed following treatment. Select patients for therapy based on an FDA-approved test. ( 1 , 2.1 ) | approved | Nov 26, 2018 | openfda |
| Lenalidomide | US FDA | Myelodysplastic Syndrome | . • Transfusion-dependent anemia due to low- or intermediate-1-risk myelodysplastic syndromes (MDS) associated with a deletion 5q abnormality with or without additional cytogenetic abnormalities | approved | Dec 27, 2005 | openfda |
| Midostaurin | US FDA | Acute Myeloid Leukemia | Newly diagnosed acute myeloid leukemia (AML) that is FLT3 mutation-positive as detected by an FDA-approved test, in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation. | approved | Apr 28, 2017 | openfda |
| Midostaurin | US FDA | Acute Myeloid Leukemia | Newly diagnosed acute myeloid leukemia (AML) that is FLT3 mutation-positive as detected by an FDA-approved test, in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation. | approved | Apr 28, 2017 | openfda |
| Nilotinib | US FDA | Chronic Myeloid Leukemia, Philadelphia Chromosome Negative, BCR-ABL1 Positive | Adult patients with chronic phase (CP) and accelerated phase (AP) Ph+ CML resistant to or intolerant to prior therapy that included imatinib. | approved | May 29, 2026 | openfda |
| Nilotinib | US FDA | Chronic Myeloid Leukemia, Philadelphia Chromosome Negative, BCR-ABL1 Positive | Adult patients with chronic phase (CP) and accelerated phase (AP) Ph+ CML resistant to or intolerant to prior therapy that included imatinib. | approved | Feb 19, 2025 | openfda |
| Nilotinib | US FDA | Chronic Myeloid Leukemia, Philadelphia Chromosome Negative, BCR-ABL1 Positive | Adult patients with chronic phase (CP) and accelerated phase (AP) Ph+ CML resistant to or intolerant to prior therapy that included imatinib. | approved | Nov 7, 2024 | openfda |
| Nilotinib | US FDA | Chronic Myeloid Leukemia, Philadelphia Chromosome Negative, BCR-ABL1 Positive | Pediatric patients greater than or equal to 1 year of age with Ph+ CML-CP and CML-AP resistant or intolerant to prior tyrosine-kinase inhibitor (TKI) therapy. | approved | Oct 29, 2007 | openfda |
| Nilotinib | US FDA | Chronic Myeloid Leukemia, Philadelphia Chromosome Negative, BCR-ABL1 Positive | Adult patients with chronic phase (CP) and accelerated phase (AP) Ph+ CML resistant to or intolerant to prior therapy that included imatinib. | approved | Oct 29, 2007 | openfda |
| Olutasidenib | US FDA | Acute Myeloid Leukemia | Relapsed or Refractory Acute Myeloid Leukemia REZLIDHIA is indicated for the 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 [see Dosage and Administration ( 2.1 ), Clinical Pharmacology ( 12.1 ), and Clinical Studies ( 14.1 )] . REZLIDHIA is an isocitrate dehydrogenase-1 (IDH1) inhibitor indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible IDH1 mutation as detected by an FDA-approved test. ( 1 ) | approved | Dec 1, 2022 | openfda |
| Pembrolizumab | US FDA | Tumor-agnostic | for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy. • Limitations of Use : KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy. Urothelial Cancer in combination with enfortumab vedotin-ejfv, for the treatment of adult patients with locally advanced or metastatic urothelial cancer. • Microsatellite Instability-High or Mismatch Repair Deficient Cancer for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors, as determined by an FDA-authorized test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. • in combination with fluoropyrimidine- and platinum-containing chemotherapy, for the first-line treatment of adults with locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-authorized test. • in combination with lenvatinib, for the first-line treatment of adult patients with advanced RCC. • for the adjuvant treatment of patients with RCC at intermediate-high or high risk of recurrence following nephrectomy, or following nephrectomy and resection of metastatic lesions. • Tumor Mutational Burden-High (TMB-H) Cancer for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase (mut/Mb)] solid tumors, as determined by an FDA-authorized test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. 1 • Limitations of Use : The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established. Cutaneous Squamous Cell Carcinoma (cSCC) for the treatment of patients with recurrent or metastatic cSCC or locally advanced cSCC that is not curable by surgery or radiation. • Ovarian Cancer in combination with paclitaxel, with or without bevacizumab, for the treatment of adult patients with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal carcinoma whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-authorized test, and who have received one or two prior systemic treatment regimens. • 1 This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.accelerated | approved |
| Quizartinib | US FDA | Acute Myeloid Leukemia | VANFLYTA is indicated 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 [see Dosage and Administration (2.1) and Clinical Studies (14) ] . VANFLYTA is a kinase inhibitor indicated 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. ( 1 ) Limitations of Use: VANFLYTA is not indicated as maintenance monotherapy following allogeneic hematopoietic stem cell transplantation (HSCT); improvement in overall survival with VANFLYTA in this setting has not been demonstrated. ( 1 ) Limitations of Use VANFLYTA is not indicated as maintenance monotherapy following allogeneic hematopoietic stem cell transplantation (HSCT); improvement in overall survival with VANFLYTA in this setting has not been demonstrated [see Clinical Studies (14) ] . | approved | Jul 20, 2023 | openfda |
| Repotrectinib | US FDA | Tumor-agnostic | adult and pediatric patients 12 years of age and older with solid tumors that: • have a neurotrophic tyrosine receptor kinase ( NTRK ) gene fusion and • are locally advanced or metastatic or where surgical resection is likely to result in severe morbidity. • have progressed following treatment or have no satisfactory alternative therapy. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.accelerated | approved | Nov 15, 2023 | openfda |
| Revumenib | US FDA | Acute Myeloid Leukemia | REVUFORJ is a menin inhibitor indicated for: the treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene ( KMT2A ) translocation as determined by an FDA-authorized test in adult and pediatric patients 1 year and older. ( 1 ) the treatment of relapsed or refractory acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 ( NPM1 ) mutation in adult and pediatric patients 1 year and older who have no satisfactory alternative treatment options. ( 1 ) Relapsed or Refractory Acute Leukemia REVUFORJ is indicated for the treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene ( KMT2A ) translocation as determined by an FDA-authorized test in adult and pediatric patients 1 year and older. REVUFORJ is indicated for the treatment of relapsed or refractory acute myeloid leukemia with a susceptible nucleophosmin 1 ( NPM1 ) mutation [see Dosage and Administration (2.1) , Clinical Pharmacology (12.1) , and Clinical Studies (14.1) ] in adult and pediatric patients 1 year and older who have no satisfactory alternative treatment options. | approved | Nov 15, 2024 | openfda |
| Ropeginterferon Alfa-2b | US FDA | Essential Thrombocythemia | The treatment of adults with essential thrombocythemia. | approved | Nov 12, 2021 | openfda |
| Ropeginterferon Alfa-2b | US FDA | Polycythemia Vera | The treatment of adults with polycythemia vera. | approved | Nov 12, 2021 | openfda |
| Ruxolitinib | US FDA | Polycythemia Vera | polycythemia vera in adults who have had an inadequate response to or are intolerant of hydroxyurea. | approved | May 1, 2026 | openfda |
| Ruxolitinib | US FDA | Polycythemia Vera | polycythemia vera in adults who have had an inadequate response to or are intolerant of hydroxyurea. | approved | Nov 16, 2011 | openfda |
| Selpercatinib | US FDA | Tumor-agnostic | Adult and pediatric patients 2 years of age and older with locally advanced or metastatic solid tumors with a RET gene fusion, as detected by an FDA-approved test, that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options | approved | Apr 10, 2024 | openfda |
| Selpercatinib | US FDA | Tumor-agnostic | Adult and pediatric patients 2 years of age and older with locally advanced or metastatic solid tumors with a RET gene fusion, as detected by an FDA-approved test, that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options | approved | May 8, 2020 | openfda |
| Trametinib | US FDA | Tumor-agnostic | the treatment of adult and pediatric patients 1 year of age and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment and have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). • the treatment of pediatric patients 1 year of age and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy.accelerated | approved | Mar 16, 2023 | openfda |