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Total: 23
Small molecule
Approved
HEMATOLOGY; OTHER
ORAL
Hypertension, Pulmonary; Lung Neoplasms; Paraganglioma; beta-Thalassemia; Central Nervous System Neoplasms; Desmoid Tumors; Esophageal Neoplasms; Fibroma; Gliosarcoma; Head and Neck Neoplasms; Hemoglobin SC Disease; Leukemia, Myeloid, Acute; Lymphoma; Meningioma; Multiple Sclerosis; Muscular Atrophy, Spinal; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Stomach Neoplasms; Thalassemia; Astrocytoma; Carcinoma; Glioblastoma; Hematologic Diseases; Hemochromatosis; Hemoglobinopathies; HIV Infections; Laryngeal Neoplasms; Leukemia; Leukemia, Promyelocytic, Acute; Mouth Neoplasms; Myelodysplastic Syndromes; Myeloproliferative Disorders; Paranasal Sinus Neoplasms; Pharyngeal Neoplasms; Polycythemia Vera; Primary Myelofibrosis; Stroke; Thrombocythemia, Essential; Anemia, Sickle Cell; Carcinoma, Squamous Cell; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Melanoma; Neoplasms
Ribonucleoside-diphosphate reductase RR1 inhibitor
Small molecule
Approved
DIABETES DRUGS & ENDOCRINOLOGY; HEMATOLOGY; OTHER
ORAL
Angina, Stable; Ischemic Stroke; Spinal Cord Injuries; Brain Injuries; Diabetes Mellitus, Type 1; Stroke; Diabetes, Gestational; Diabetes Mellitus; Diabetes Mellitus, Type 2
Sulfonylurea receptor 1, Kir6.2 blocker
Protein
Approved
CANCER; HEMATOLOGY; OTHER
UNKNOWN
Alzheimer Disease; beta-Thalassemia; Blast Crisis; Carcinoma, Endometrioid; Chordoma; Coronary Disease; Cystadenoma, Serous; Enterocolitis, Necrotizing; Ganglioneuroblastoma; Glioma; Granulomatous Disease, Chronic; Hemangiosarcoma; Immunologic Deficiency Syndromes; Leukemia, Plasma Cell; Leukemia, Promyelocytic, Acute; Lymphoma, B-Cell, Marginal Zone; Menopause; Multiple Sclerosis; Muscular Diseases; Myasthenia Gravis; Neoplasm Metastasis; Neurilemmoma; Peripheral Arterial Disease; Peripheral Vascular Diseases; Primary Myelofibrosis; Sarcoma, Synovial; Severe Combined Immunodeficiency; Stiff-Person Syndrome; Vasculitis; Waldenstrom Macroglobulinemia; Amyotrophic Lateral Sclerosis; Anemia, Diamond-Blackfan; Anemia, Sickle Cell; Biliary Atresia; Breast Neoplasms, Male; Carcinoid Tumor; Carcinoma; Carcinoma, Hepatocellular; Carcinoma, Islet Cell; Carcinoma, Large Cell; Carcinoma, Renal Cell; Carcinoma, Squamous Cell; Cardiomyopathy, Dilated; Cerebral Palsy; Colorectal Neoplasms; Crohn Disease; Diabetes Mellitus, Type 1; Endometriosis; Epidermolysis Bullosa Dystrophica; Esophageal Neoplasms; Fallopian Tube Neoplasms; Fanconi Anemia; Gallbladder Neoplasms; Glioblastoma; Gliosarcoma; Hematologic Diseases; Hematologic Neoplasms; Histiocytosis, Langerhans-Cell; HIV Infections; Infertility; Inflammatory Breast Neoplasms; Ischemic Stroke; Leukemia, Mast-Cell; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Leukemia, Myeloid; Liver Neoplasms; Lupus Erythematosus, Systemic; Lymphoma, AIDS-Related; Lymphoma, Large-Cell, Anaplastic; Lymphoma, T-Cell; Lymphoma, T-Cell, Peripheral; Lymphopenia; Myelodysplastic-Myeloproliferative Diseases; Nasopharyngeal Carcinoma; Neuromyelitis Optica; Osteosarcoma; Pancreatic Neoplasms; Parkinson Disease; Pemphigus; Peritoneal Neoplasms; Prostatic Neoplasms; Sarcoma, Ewing; Small Cell Lung Carcinoma; Stomach Neoplasms; Stroke; Urethral Neoplasms; Uterine Cervical Neoplasms; Wilms Tumor; Adrenal Cortex Neoplasms; Amyloidosis, Familial; Anemia; Anemia, Aplastic; Anemia, Refractory; Anemia, Refractory, with Excess of Blasts; Brain Neoplasms; Breast Neoplasms; Burkitt Lymphoma; Carcinoma, Non-Small-Cell Lung; Central Nervous System Neoplasms; Chagas Cardiomyopathy; Endometrial Neoplasms; Ependymoma; Fever; Graft vs Host Disease; Head and Neck Neoplasms; Hepatitis, Alcoholic; Hodgkin Disease; Hot Flashes; Kidney Neoplasms; Leukemia; Leukemia, Basophilic, Acute; Leukemia, Biphenotypic, Acute; Leukemia, Erythroblastic, Acute; Leukemia, Lymphocytic, Chronic, B-Cell; Leukemia-Lymphoma, Adult T-Cell; Leukemia, Megakaryoblastic, Acute; Leukemia, Monocytic, Acute; Leukemia, Myeloid, Acute; Leukemia, Myelomonocytic, Acute; Leukemia, Myelomonocytic, Chronic; Liver Cirrhosis; Liver Diseases; Liver Failure; Lung Neoplasms; Lymphoma; Lymphoma, Follicular; Lymphoma, Large B-Cell, Diffuse; Lymphoma, Mantle-Cell; Lymphoma, Non-Hodgkin; Mediastinal Neoplasms; Medulloblastoma; Melanoma; Multiple Myeloma; Multiple Sclerosis, Relapsing-Remitting; Myelodysplastic Syndromes; Myeloproliferative Disorders; Myocardial Infarction; Neoplasms, Germ Cell and Embryonal; Neoplasms, Plasma Cell; Neuroblastoma; Neuroectodermal Tumors, Primitive; Ovarian Neoplasms; Pheochromocytoma; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Primary Ovarian Insufficiency; Prostatic Neoplasms, Castration-Resistant; Retinoblastoma; Rhabdomyosarcoma; Sarcoma; Scleroderma, Systemic; ST Elevation Myocardial Infarction; Testicular Neoplasms; Triple Negative Breast Neoplasms; Urinary Bladder Neoplasms; Uterine Neoplasms; Hematopoietic Stem Cell Transplantation; Infections; Neoplasms; Neutropenia
Granulocyte colony stimulating factor receptor agonist
Protein
Approved
DIABETES DRUGS & ENDOCRINOLOGY; HEMATOLOGY; OTHER
UNKNOWN
Adenocarcinoma; Anemia, Refractory, with Excess of Blasts; Carcinoma, Pancreatic Ductal; Hepatitis B, Chronic; Leukemia, Plasma Cell; Liver Neoplasms; Neoplasm Metastasis; Parkinson Disease; Periodontitis; Sepsis; Alzheimer Disease; Biliary Tract Neoplasms; Brain Neoplasms; Breast Neoplasms, Male; Carcinoma, Endometrioid; Carcinoma, Intraductal, Noninfiltrating; Carcinoma, Non-Small-Cell Lung; Carcinoma, Renal Cell; Carcinoma, Squamous Cell; Central Nervous System Neoplasms; Colorectal Neoplasms; Cystadenoma, Serous; Down Syndrome; Esophageal Neoplasms; Fallopian Tube Neoplasms; Gallbladder Neoplasms; Glioblastoma; Gliosarcoma; Hematologic Neoplasms; HIV Infections; Hodgkin Disease; Inflammatory Breast Neoplasms; Kidney Diseases; Kidney Neoplasms; Leishmaniasis, Cutaneous; Leukemia, Lymphocytic, Chronic, B-Cell; Lung Neoplasms; Lymphoma, Follicular; Myelodysplastic-Myeloproliferative Diseases; Myeloproliferative Disorders; Neoplasms, Plasma Cell; Osteosarcoma; Ovarian Neoplasms; Peritoneal Neoplasms; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Prostatic Neoplasms; Prostatic Neoplasms, Castration-Resistant; Pulmonary Alveolar Proteinosis; Severe Acute Respiratory Syndrome; Squamous Cell Carcinoma of Head and Neck; Stomach Neoplasms; Triple Negative Breast Neoplasms; Breast Neoplasms; Crohn Disease; Cytomegalovirus Retinitis; Ganglioneuroblastoma; Infections; Leukemia; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Lymphoma; Lymphoma, Non-Hodgkin; Melanoma; Multiple Myeloma; Myelodysplastic Syndromes; Neuroblastoma; Pancreatic Neoplasms; Rhabdomyosarcoma; Sarcoma; Urinary Bladder Neoplasms; Uveal Melanoma; Leukemia, Myeloid, Acute; Neoplasms
Granulocyte-macrophage colony-stimulating factor receptor agonist
Antibody
Approved
HEMATOLOGY; OTHER
UNKNOWN
Arthritis, Psoriatic; Autoimmune Diseases; Carcinoma, Squamous Cell; Crohn Disease; Encephalomyelitis; Fanconi Anemia; Gangliosidoses; Glycogen Storage Disease Type II; Gout; Graves Disease; Hematologic Diseases; Liver Cirrhosis, Biliary; Lymphoma, Primary Effusion; Lymphoma, T-Cell, Cutaneous; Macular Degeneration; Muscular Diseases; Mycosis Fungoides; Neuroblastoma; Opsoclonus-Myoclonus Syndrome; Pemphigoid, Benign Mucous Membrane; Prostatic Neoplasms, Castration-Resistant; Renal Insufficiency, Chronic; Scleritis; Urticaria; Acquired Immunodeficiency Syndrome; Anemia, Aplastic; Anemia, Sickle Cell; Behcet Syndrome; Blast Crisis; Blastic Plasmacytoid Dendritic Cell Neoplasm; Breast Neoplasms; Bronchiolitis Obliterans Syndrome; Carcinoma, Hepatocellular; Castleman Disease; Churg-Strauss Syndrome; Colitis, Ulcerative; Colonic Neoplasms; Dermatomyositis; Diabetes Mellitus, Type 1; Diabetic Neuropathies; Encephalitis, Japanese; Glomerulonephritis; Glomerulosclerosis, Focal Segmental; Graves Ophthalmopathy; Heart Failure; Hematologic Neoplasms; Hemolytic-Uremic Syndrome; Hemophilia A; Histiocytosis, Langerhans-Cell; HTLV-I Infections; Idiopathic Pulmonary Fibrosis; Immunoblastic Lymphadenopathy; Kidney Failure, Chronic; Leukemia, Biphenotypic, Acute; Leukemia, Hairy Cell; Leukemia, Mast-Cell; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Leukemia, Myeloid, Acute; Leukemia, Myelomonocytic, Chronic; Leukemia, Prolymphocytic; Lung Neoplasms; Lymphoma, AIDS-Related; Lymphoma, B-Cell; Lymphoma, Large-Cell, Anaplastic; Lymphoproliferative Disorders; Melanoma; Multiple Myeloma; Multiple Sclerosis, Chronic Progressive; Myelodysplastic Syndromes; Neoplasms, Plasma Cell; Neuromyelitis Optica; Pancreatic Neoplasms; Peripheral Nervous System Diseases; Pneumococcal Infections; Polyendocrinopathies, Autoimmune; Precursor T-Cell Lymphoblastic Leukemia-Lymphoma; Psychotic Disorders; Pulmonary Alveolar Proteinosis; Renal Insufficiency; Rheumatic Heart Disease; Sarcoidosis; Schizophrenia; Scleroderma, Systemic; Sjogren's Syndrome; Spinal Cord Diseases; Spondylitis, Ankylosing; Stiff-Person Syndrome; Stomach Neoplasms; Virus Diseases; Anemia, Hemolytic, Autoimmune; Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis; Burkitt Lymphoma; Fatigue Syndrome, Chronic; Glomerulonephritis, Membranoproliferative; Glomerulonephritis, Membranous; Graft vs Host Disease; Hodgkin Disease; IgA Vasculitis; Immune System Diseases; Kidney Diseases; Leukemia; Leukemia, Lymphoid; Lung Diseases; Lung Diseases, Interstitial; Lupus Erythematosus, Systemic; Lupus Nephritis; Lymphoma; Lymphoma, B-Cell, Marginal Zone; Lymphoma, Follicular; Lymphoma, Large B-Cell, Diffuse; Lymphoma, Mantle-Cell; Mental Disorders; Multiple Sclerosis; Multiple Sclerosis, Relapsing-Remitting; Myasthenia Gravis; Nephrotic Syndrome; Pemphigoid, Bullous; Polymyalgia Rheumatica; Polyneuropathies; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating; Proteinuria; Purpura, Thrombocytopenic, Idiopathic; Purpura, Thrombotic Thrombocytopenic; Waldenstrom Macroglobulinemia; Arthritis, Rheumatoid; Granulomatosis with Polyangiitis; Leukemia, Lymphocytic, Chronic, B-Cell; Lymphoma, Non-Hodgkin; Microscopic Polyangiitis; Neoplasms; Pemphigus; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Systemic Vasculitis
B-lymphocyte antigen CD20 binding agent
Small molecule
Approved
HEMATOLOGY
ORAL
Cerebellar Ataxia; Cognitive Dysfunction; HIV Infections; Myocardial Infarction; Amyotrophic Lateral Sclerosis; Friedreich Ataxia; Parkinson Disease; Stroke; Acute Kidney Injury; Hemochromatosis; Pantothenate Kinase-Associated Neurodegeneration; Anemia, Sickle Cell; beta-Thalassemia; Iron Overload; Thalassemia
Unknown
Small molecule
Approved
HEMATOLOGY; OTHER; UROLOGY AND KIDNEY
INTRAVENOUS
Hematologic Neoplasms; Hypertension, Pulmonary; Anemia; Anemia, Iron-Deficiency; Renal Insufficiency, Chronic
Supplement
Small molecule
Approved
CARDIOVASCULAR; HEMATOLOGY; OTHER
INJECTION; INTRAVENOUS
Angina, Unstable; Coronary Disease; Hemorrhagic Disorders; Ischemic Stroke; Thrombocytopenia; Thrombosis
Thrombin inhibitor
Small molecule
Approved
HEMATOLOGY; OTHER
INTRAVENOUS; SUBCUTANEOUS
Acute Kidney Injury; Blastic Plasmacytoid Dendritic Cell Neoplasm; Brain Neoplasms; Carcinoma, Pancreatic Ductal; Carcinoma, Renal Cell; Carcinoma, Squamous Cell; Colorectal Neoplasms; Ependymoma; Esophageal Neoplasms; Glioma; Graft vs Host Disease; Head and Neck Neoplasms; Leukemia, Biphenotypic, Acute; Leukemia, Large Granular Lymphocytic; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Liposarcoma; Lymphoma, Non-Hodgkin; Mastocytosis, Systemic; Myelodysplastic-Myeloproliferative Diseases; Osteosarcoma; Sarcoma; Tuberculosis; beta-Thalassemia; Breast Neoplasms; Breast Neoplasms, Male; Carcinoma, Non-Small-Cell Lung; Colonic Neoplasms; Hematologic Neoplasms; Hodgkin Disease; Leukemia, Erythroblastic, Acute; Leukemia, Lymphocytic, Chronic, B-Cell; Leukemia, Lymphoid; Leukemia, Monocytic, Acute; Lymphoma, Follicular; Melanoma; Multiple Myeloma; Myeloproliferative Disorders; Nasopharyngeal Neoplasms; Ovarian Neoplasms; Pancreatic Neoplasms; Paraganglioma; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Primary Myelofibrosis; Prostatic Neoplasms; Prostatic Neoplasms, Castration-Resistant; Rectal Neoplasms; Immunoblastic Lymphadenopathy; Leukemia; Leukemia, Myeloid; Lymphoma; Lymphoma, Large B-Cell, Diffuse; Lymphoma, T-Cell, Peripheral; Thrombocytopenia; Anemia, Refractory, with Excess of Blasts; Leukemia, Myeloid, Acute; Leukemia, Myelomonocytic, Chronic; Myelodysplastic Syndromes; Neoplasms
DNA (cytosine-5)-methyltransferase 1 inhibitor
Small molecule
Approved
CANCER; HEMATOLOGY; OTHER
ORAL
Astrocytoma; Burkitt Lymphoma; Cachexia; Carcinoma, Pancreatic Ductal; Carcinoma, Renal Cell; Central Nervous System Neoplasms; Colonic Neoplasms; Diffuse Intrinsic Pontine Glioma; Fallopian Tube Neoplasms; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Lymphoma, B-Cell; Lymphoma, Primary Effusion; Neoplasms; Neuroblastoma; Ovarian Neoplasms; Peritoneal Neoplasms; Rectal Neoplasms; Sarcoma, Kaposi; Urethral Neoplasms; Autistic Disorder; Carcinoma, Hepatocellular; Carcinoma, Non-Small-Cell Lung; Carcinoma, Squamous Cell; Colorectal Neoplasms; Complex Regional Pain Syndromes; Crohn Disease; Glioma; Gliosarcoma; Graft vs Host Disease; Hematologic Diseases; Hematologic Neoplasms; Histiocytosis, Sinus; HIV Infections; Hodgkin Disease; Immunoglobulin Light-chain Amyloidosis; Kidney Neoplasms; Leukemia; Leukemia, Biphenotypic, Acute; Leukemia, Lymphoid; Leukemia-Lymphoma, Adult T-Cell; Leukemia, Myelomonocytic, Chronic; Leukemia, Plasma Cell; Leukemia, Prolymphocytic; Liver Neoplasms; Lymphoma, T-Cell; Lymphoma, T-Cell, Cutaneous; Lymphoma, T-Cell, Peripheral; Myeloproliferative Disorders; Pancreatic Neoplasms; POEMS Syndrome; Polycythemia Vera; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Primary Myelofibrosis; Prostatic Neoplasms; Radiculopathy; Thrombocythemia, Essential; Thyroid Neoplasms; Urinary Bladder Neoplasms; Waldenstrom Macroglobulinemia; Amyloidosis; Amyloidosis, Familial; Epstein-Barr Virus Infections; Leukemia, Lymphocytic, Chronic, B-Cell; Leukemia, Myeloid, Acute; Lymphoma; Lymphoma, B-Cell, Marginal Zone; Lymphoma, Follicular; Lymphoma, Large B-Cell, Diffuse; Lymphoma, Non-Hodgkin; Melanoma; Neoplasm Metastasis; Neoplasms, Plasma Cell; Plasmacytoma; Prostatic Neoplasms, Castration-Resistant; Smoldering Multiple Myeloma; Anemia; Immune System Diseases; Lymphoma, Mantle-Cell; Multiple Myeloma; Myelodysplastic Syndromes
CRL4(CRBN) E3 ubiquitin ligase inhibitor
Small molecule
Approved
HEMATOLOGY; OTHER
INTRAVENOUS
Burkitt Lymphoma; Carcinoma, Squamous Cell; Head and Neck Neoplasms; Hematologic Neoplasms; HIV Infections; Leukemia, Promyelocytic, Acute; Lymphoma, Follicular; Lymphoma, Mantle-Cell; Lymphoma, Non-Hodgkin; Lymphoma, T-Cell, Cutaneous; Mastocytosis, Systemic; Medulloblastoma; Multiple Myeloma; Neuroblastoma; Paraganglioma; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Precursor T-Cell Lymphoblastic Leukemia-Lymphoma; Prostatic Neoplasms; Sarcoma, Synovial; Triple Negative Breast Neoplasms; Adenocarcinoma, Follicular; Anemia, Aplastic; Anemia, Sickle Cell; Breast Neoplasms; Carcinoma, Non-Small-Cell Lung; Carcinoma, Renal Cell; Esophageal Neoplasms; Fallopian Tube Neoplasms; Granuloma, Lethal Midline; Hodgkin Disease; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Lung Neoplasms; Lymphoma; Melanoma; Ovarian Neoplasms; Peritoneal Neoplasms; Primary Myelofibrosis; Severe Acute Respiratory Syndrome; Thalassemia; Thyroid Neoplasms; Graft vs Host Disease; Hematologic Diseases; Leukemia; Leukemia, Myeloid, Acute; Lymphoma, Large B-Cell, Diffuse; Lymphoma, T-Cell, Peripheral; Myeloproliferative Disorders; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia; Anemia; Anemia, Refractory, with Excess of Blasts; Leukemia, Myeloid; Leukemia, Myelomonocytic, Chronic; Myelodysplastic Syndromes; Neoplasms
DNA (cytosine-5)-methyltransferase 1 inhibitor
Antibody
Approved
HEMATOLOGY
IV (INFUSION)
End Stage Liver Disease; Graft vs Host Disease; Hemolysis; Anemia, Hemolytic, Autoimmune; Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis; Dermatomyositis; Diabetes Mellitus; Glomerulonephritis, Membranoproliferative; Macular Degeneration; Renal Insufficiency, Chronic; Severe Acute Respiratory Syndrome; Thrombocytopenia; Guillain-Barre Syndrome; Hemolytic-Uremic Syndrome; Leukemia; Myasthenia Gravis; Neuromyelitis Optica; Atypical Hemolytic Uremic Syndrome; Hemoglobinuria; Hemoglobinuria, Paroxysmal
Complement C5 inhibitor
Small molecule
Approved
HEMATOLOGY; OTHER
ORAL
Lymphoma; Myelodysplastic Syndromes; Anemia, Aplastic; Hepatitis C, Chronic; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia
Thrombopoietin receptor agonist
Protein
Approved
HEMATOLOGY
SUBCUTANEOUS
Lymphoma; Neoplasms; Purpura, Thrombocytopenic; Anemia, Aplastic; Carcinoma; Glioblastoma; Lymphoma, Non-Hodgkin; Myelodysplastic Syndromes; Ovarian Neoplasms; Purpura, Thrombotic Thrombocytopenic; Wiskott-Aldrich Syndrome; Hemorrhage; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia
Thrombopoietin receptor agonist
Small molecule
Approved
HEMATOLOGY; OTHER
ORAL
Carcinoma; Kidney Diseases; Kidney Failure, Chronic; Dyspnea; Edema, Cardiac; Liver Cirrhosis; Cardiovascular Diseases; Heart Failure; Hyponatremia; Inappropriate ADH Syndrome; Polycystic Kidney, Autosomal Dominant; Polycystic Kidney Diseases
Vasopressin V2 receptor antagonist
Protein
Approved
HEMATOLOGY
INTRAVENOUS
Astrocytoma; Brain Neoplasms; Carcinoma, Pancreatic Ductal; Glioblastoma; Gliosarcoma; Hematologic Neoplasms; Hodgkin Disease; Inflammatory Breast Neoplasms; Leukemia; Liposarcoma; Lymphoma, Non-Hodgkin; Mycosis Fungoides; Oligodendroglioma; Pancreatic Neoplasms; Sezary Syndrome; Triple Negative Breast Neoplasms; Adenocarcinoma, Follicular; Breast Neoplasms; Breast Neoplasms, Male; Carcinoma, Non-Small-Cell Lung; Colorectal Neoplasms; HIV Infections; Leukemia, Lymphocytic, Chronic, B-Cell; Leukemia, Promyelocytic, Acute; Lymphoma, Follicular; Lymphoma, Large B-Cell, Diffuse; Lymphoma, Large-Cell, Anaplastic; Lymphoma, Mantle-Cell; Melanoma; Multiple Myeloma; Ovarian Neoplasms; Prostatic Neoplasms, Castration-Resistant; Small Cell Lung Carcinoma; Stomach Neoplasms; Thyroid Neoplasms; Urethral Neoplasms; Urinary Bladder Neoplasms; Immunoblastic Lymphadenopathy; Lymphoma; Lymphoma, T-Cell; Lymphoma, T-Cell, Cutaneous; Lymphoma, T-Cell, Peripheral; Neoplasms
Histone deacetylase inhibitor
Small molecule
Approved
CARDIOVASCULAR; HEMATOLOGY; OTHER
ORAL
Blood Coagulation Disorders; Obesity, Morbid; Renal Insufficiency, Chronic; Anemia, Sickle Cell; Aortic Valve Disease; Heart Diseases; Hemorrhage; Ischemia; Multiple Myeloma; Thromboembolism; Angina, Unstable; Aortic Valve Stenosis; Diabetes Mellitus, Type 2; Hematoma; Hip Fractures; Ischemic Stroke; Liver Cirrhosis; Myocardial Infarction; Neoplasms; Polyendocrinopathies, Autoimmune; Renal Insufficiency; Rheumatic Heart Disease; Severe Acute Respiratory Syndrome; Thrombocytopenia; Acute Coronary Syndrome; Arthroplasty, Replacement; Atrial Fibrillation; Coronary Artery Disease; Peripheral Arterial Disease; Pulmonary Embolism; Stroke; Thrombosis; Venous Thromboembolism; Venous Thrombosis
Coagulation factor X inhibitor
Enzyme
Approved
HEMATOLOGY
INTRAVENOUS
Gaucher Disease
Glucocerebroside hydrolytic enzyme
Small molecule
Approved
HEMATOLOGY
ORAL
Breast Neoplasms; Carcinoma, Renal Cell; Colonic Neoplasms; Colorectal Neoplasms; Glioblastoma; Liver Diseases; Lung Neoplasms; Myelodysplastic Syndromes; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Amyloidosis; Anaphylaxis; Anemia, Hemolytic, Autoimmune; Burkitt Lymphoma; Carcinoid Tumor; Carcinoma, Non-Small-Cell Lung; Carcinoma, Squamous Cell; Esophageal Neoplasms; Food Hypersensitivity; Head and Neck Neoplasms; Hematologic Neoplasms; Hodgkin Disease; Leukemia; Leukemia, Lymphoid; Leukemia, Mast-Cell; Leukemia, Myeloid, Acute; Leukemia, Prolymphocytic; Leukemia, Prolymphocytic, T-Cell; Mastocytosis, Systemic; Melanoma; Multiple Myeloma; Prostatic Neoplasms, Castration-Resistant; Severe Acute Respiratory Syndrome; Bronchiolitis Obliterans Syndrome; Graft vs Host Disease; Lymphoma; Lymphoma, B-Cell, Marginal Zone; Lymphoma, Follicular; Lymphoma, Large B-Cell, Diffuse; Lymphoma, Non-Hodgkin; Leukemia, Lymphocytic, Chronic, B-Cell; Lymphoma, Mantle-Cell; Neoplasms; Waldenstrom Macroglobulinemia
Tyrosine-protein kinase BTK inhibitor
Small molecule
Approved
CANCER; HEMATOLOGY; OTHER
ORAL
Anemia, Sickle Cell; Burkitt Lymphoma; Castleman Disease; Hodgkin Disease; Immunoglobulin Light-chain Amyloidosis; Lymphoma; Lymphoma, Non-Hodgkin; Neoplasms; Pancreatic Neoplasms; Small Cell Lung Carcinoma; Waldenstrom Macroglobulinemia; Amyloidosis; Amyloidosis, Familial; Central Nervous System Neoplasms; Graft vs Host Disease; Leukemia, Plasma Cell; Lung Diseases, Interstitial; Lymphoma, Large B-Cell, Diffuse; Medulloblastoma; Myeloproliferative Disorders; Neoplasms, Plasma Cell; Plasmacytoma; Polycythemia Vera; Prostatic Neoplasms, Castration-Resistant; Pulmonary Fibrosis; Sarcoma; Sarcoma, Kaposi; Scleroderma, Systemic; Smoldering Multiple Myeloma; Telangiectasia, Hereditary Hemorrhagic; Primary Myelofibrosis; Immune System Diseases; Multiple Myeloma
CRL4(CRBN) E3 ubiquitin ligase inhibitor
Small molecule
Approved
HEMATOLOGY
INTRAVENOUS
Adenocarcinoma; Blast Crisis; Carcinoma, Non-Small-Cell Lung; Hematologic Neoplasms; Hodgkin Disease; Leukemia; Liver Neoplasms; Lymphoma; Lymphoma, Follicular; Mycosis Fungoides; Sarcoma; Small Cell Lung Carcinoma; Urinary Bladder Neoplasms; Burkitt Lymphoma; Carcinoma; Carcinoma, Endometrioid; Cystadenoma, Serous; Fallopian Tube Neoplasms; Glioblastoma; Leukemia-Lymphoma, Adult T-Cell; Leukemia, Myeloid, Acute; Lymphoma, Large B-Cell, Diffuse; Lymphoma, Large-Cell, Anaplastic; Lymphoma, Mantle-Cell; Lymphoma, Non-Hodgkin; Lymphoma, T-Cell, Cutaneous; Mesothelioma; Multiple Myeloma; Myelodysplastic Syndromes; Peritoneal Neoplasms; Thymoma; Uveal Melanoma; Lymphoma, T-Cell; Lymphoma, T-Cell, Peripheral; Neoplasms
Histone deacetylase inhibitor
Small molecule
Approved
CANCER; HEMATOLOGY; OTHER
ORAL
Carcinoma, Pancreatic Ductal; Leukemia, Myeloid, Acute; Multiple Myeloma; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Primary Myelofibrosis; Rhinitis, Allergic, Seasonal; Amyloidosis; Hodgkin Disease; Lymphoma, Large B-Cell, Diffuse; Lymphoma, Mantle-Cell; Waldenstrom Macroglobulinemia; Lymphoma; Lymphoma, Follicular; Leukemia, Lymphocytic, Chronic, B-Cell; Lymphoma, Non-Hodgkin; Neoplasms
PI3-kinase p110-delta subunit inhibitor
Small molecule
Approved
CANCER; HEMATOLOGY; OTHER
ORAL
Leukemia, Myeloid, Acute; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Leukemia, Myeloid
Bcr/Abl fusion protein inhibitor