The ChEMBL-og

ATC Code: L01XX47 Wikipedia: Idelalisib ChEMBL: CHEMBL2216870 On July 23rd the FDA approved Zydelig ( idelalisib , GS-1101), as an orally-delivered drug to treat patients with three types of blood cancers. • Relapsed chronic lymphocytic leukemia (CLL) • Relapsed follicular B-cell, non-Hodgkin lymphoma  (FL) • Relapsed small lymphocytic lymphoma (SLL) Blood cancer The three main categories of blood cancer are leukemia , lymphoma and myeloma . Lymphoma is also split into two types: Hodgkin lymphoma and non-Hodgkin lymphoma . Both leukemia and myeloma occur in the bone marrow , whilst lymphoma is a cancer that is isolated to the lymphatic system. Acute leukemia is where there is an abundance of underdeveloped white blood cells that can’t function properly and chronic leukemia is where there are just far too many white blood cells, which is just as bad as having too few. Myeloma is where the plasma cells form tumours in the bone marrow. ...

Wikipedia : Albiglutide ChEMBL :  CHEMBL2107841 On April 15th the FDA approved Tanzeum (albiglutide) subcutaneous injection to improve glycemic control, along with diet and exercise, in adults with type 2 diabetes. Type II diabetes Type II diabetes is a metabolic disorder that is characterized by high blood sugar (hyperglycemia) due to insulin resistance or relative lack of insulin. The disease affects millions of patient world-wide and can lead to long-term complications if the blood levels are not lowered in the patients: heart diseases, strokes and kidney failure. Albiglutide The drug is a dipeptidyl peptidase-4-resistant glucagon-like peptide-1 dimer fused to human albumin. Schematic representation of the albiglutide ( EMA ) Mode of action Traditionally, a decrease in the glucose blood level of affected patients is triggered using insulin injections. One alternative mechanism consists at indirectly stimulating insulin release us...

ATC Code: Wikipedia: Ramucirumab ChEMBL: CHEMBL1743062 On April 21, 2014 the FDA approved Ramucirumab (Cyramza™) for the treatment of patients with advanced or metastatic, gastric or gastroesophageal junction (GEJ) adenocarcinoma with disease progression on or after prior treatment with fluoropyrimidine- or platinum-containing chemotherapy. Gastric cancer has a very poor prognosis, with adenocarcinomas constituting ~95% of all gastric cancers ( CRUK ). In a randomized, double-blind, multicenter study of ramucirumab plus best supportive care (BSC) compared with placebo plus BSC of 355 patients with locally advanced or metastatic gastric cancer (including adenocarcinoma of the gastro-esophageal junction [GEJ]), ramucirumab improved the overall survival to a median of 5.2 months, compared to 3.8 with the placebo arm. Progression-free survival (PFS) was improved from a median of 1.3 months in the placebo arm to 2.1 months in the ramucirumab arm. Cyramza has been is...

ATC Code:  Unavailable Wikipedia:   Florbetaben_F18 ChEMBL:  CHEMBL1908906 On March 19th the FDA approved  Florbetaben F18 (Neuraceq™) as a radioactive diagnostic agent for Positron Emission Tomography (PET) imaging of the brain to estimate β-amyloid (βA) neuritic plaque density in adult patients with cognitive impairment who are being evaluated for Alzheimer’s disease or other causes of cognitive decline. Alzheimer's disease is the most common form of dementia, can currently not be cured and is characterised by a progressive disease pattern that usually leads to death.  Alzheimer's is predicted to affect 1 in 85 people globally by 2050 . Target(s) Florbetaben binds with high affinity to βA  in brain homogenates and selectively labels βA plaques and cerebral amyloid angiopathy. βA ( PDB  ; Uniprot  P05067 ) denotes 36-43 length peptides that are believed to be crucially involved in the Alzheimer's disease mechanism....

ATC Code (s): A08A , A10X , A16A Wikipedia: Metreleptin On February 24 th  2014, the FDA approved metreleptin (Tradename: Myalept ), a leptin analogue, as an adjunct to diet and replacement therapy, for the treatment of complications associated with leptin deficiency in patients with congenital or acquired generalized lipodystrophy . Lipodystrophy is a rare condition characterized by abnormalities in adipose (fat) tissue distribution. It can be congenital, i.e. the patient is born with little or no adipose tissue, or it can be acquired, for example, after prolonged antiretroviral drug therapy some patients keep on losing adipose tissue with time. The deficiency of adipose tissue leads to hypertriglyceridemia and ectopic deposition of fat in non-adipose tissues such as liver and muscle, contributing to metabolic abnormalities including insulin resistance . Leptin is an endogenous hormone, predominantly secreted in the adipose tissue, responsible to signal to the ...

ATC Code:  Unavailable Wikipedia:   Droxidopa ChEMBL:  CHEMBL2103827 On February 18th the  FDA approved  Droxidopa (tarde name Northera™) for the treatment of neurogenic orthostatic hypotension (NOH). NOH is a rare, chronic and often debilitating drop in blood pressure upon standing, and is associated with Parkinson's disease, multiple-system atrophy, and pure autonomic failure. Symptoms of NOH include dizziness, light-headedness, blurred vision, fatigue and fainting when a person stands.  Target(s) Droxidopa (also known as L-DOPS, L-threo-dihydroxyphenylserine, and SM-5688) is a prodrug which can be converted to norepinephrine (noradrenaline) by  Aromatic L-amino acid decarboxylase  ( Uniprot P20711  ;  EC 4.1.1.28 ). Norepinephrine in turn can be converted to epinephrine by  Phenylethanolamine N-methyltransferase  ( Uniprot P11086  ). Droxidopa can cross the blood brain barrier, contrary to e...

ATC code :  R03AL03 Wikipedia :  Umeclidinium bromide  (and  vilanterol ) ChEMBL :  CHEMBL1187833  (and  CHEMBL1198857 ) On December 18, 2013, the FDA approved Anoro Ellipta for the once-daily, long-term maintenance treatment of airflow obstruction in patients with obstructive pulmonary disease (COPD). Anoro is a combination of umeclidinium (62.5 mcg - more details below) and vilanterol inhalation powder (25 mcg -  already approved in a different formulation ). Ellipta is the single inhaler device: The majority of COPD cases are due to cigarette smoking and this lung disease is a leading cause of death in the United States. Patients affected by COPD experience breathing difficulties worsening with the time as well as chronic cough and chest tightness. Umeclidinium Umeclidinium (also known as umeclidinium bromide, GSK573719A and GSK573719) is a small molecule with a molecular weight of ...

  ATC code: A16AB12 ChEMBL: CHEMBL2108676 On February 14, 2014, the FDA approved elosulfase alfa for the treatment of Mucopolysaccharidosis Type IVA (Morquio A syndrome). Elosulfase alfa is intended to replace the missing GALNS enzyme involved in an important metabolic pathway. Absence of this enzyme leads to problems with bone development, growth and mobility. Mucopolysaccharidoses comprise a group of lysosomal storage disorders caused by the deficiency of specific lysosomal enzymes required for the catabolism of glycosaminoglycans (GAG). Mucopolysaccharidosis IVA (MPS IVA, Morquio A Syndrome) is characterized by the absence or marked reduction in N-acetylgalactosamine-6-sulfatase activity. The sulfatase activity deficiency resultsin the accumulation of the GAG substrates, KS and C6S, in the lysosomal compartment of cells throughout the body. The accumulation leads to widespread cellular, tissue, and organ dysfunction. It is a rare autosomal recessive disea...

ATC Code:  L01XC15 Wikipedia: Obinutuzumab On November 1, 2013 the FDA approved obinutuzumab (Gazyva TM ) for use in combination with chlorambucil  (a nitrogen mustard alkylating agent) for the treatment of patients with previously untreated chronic lymphocytic leukemia (CLL). CLL is the most common type of Leukaemia accounting for 35% of all reported Leukaemias (See CRUK CLL page). In a randomized three-arm clinical study, the combination of obinutuzumab (in combination with chlorambucil) improved the progression-free survival (PFS) of patients to 23.0 months compared to 11.1 months for chlorambucil alone. Obinutuzumab ( CHEMBL1743048 ) is a humanized anti-CD20 monoclonal antibody of ca . 150 kDa molecular weight. Its target, the B-lymphicyte antigen CD20, is the product of the gene MS4A1 (Uniprot: P11836; ChEMBL: CHEMBL2058 ; canSAR target synopsis . The CD20 antigen is expressed on the surface of pre B- and mature B-lymphocytes. Obinutuzumab mediates B-ce...

ATC Code:  C02KX   (incomplete) Wikipedia:   Macitentan ChEMBL:  CHEMBL2103873 On October 13th the  FDA approved   Macitentan  (trade name Opsumit  ® ) for the treatment of pulmonary arterial hypertension (PAH). Macitentan is an endothelin receptor antagonist (with affinities to both Endothelin ET-A (ETA) and Endothelin ET-B (ETB) receptor subtypes, similar in mechanism of action to the previously licensed drug Bosentan , CHEMBLID957 ). Target(s) The Endothelin receptor ET-A (ETA, CHEMBLID252  ; Uniprot P25101 ) and Endothelin receptor ET-B (ETB, CHEMBLID1785  ; Uniprot P24530 ) receptors mediate a number of physiological effects via the natural peptide agonist Endothelin-1 (ET1 , CHEMBL437472  ; Uniprot P05305 ). In addition to normal roles in supporting homeostasis, these effects can include pathologies such as inflammation, vasoconstriction, fibrosis and hypertrophy. Macitentan acts as an antagoni...

ATC code: not yet assigned Wikipedia: Riociguat On October 8, 2013, the FDA approved riociguat for the treatment of patients suffering from two forms of pulmonary hypertension - chronic thromboembolic pulmonary hypertension (CTEPH), and pulmonary arterial hypertension (PAH). Pulmonary hypertension (PH) is a disease characterized by abnormally high blood pressure in the lungs, which increases the workload for the right ventricle of the heart. Some of the symptoms of PH are dizziness, shortness of breath and water deposits in the legs and joints. PH progresses slowly and can lead to severe and often fatal circulatory and respiratory complications. CTEPH is a form of PH caused by blood clots obstructing the passage of blood through the vessels in the lung, often after a pulmonary embolism has occurred. PAH on the other hand is caused by a chronic tightening or constriction of blood vessels. Riociguat ( CHEMBL2107834 ) is a stimulator of soluble guanylate cyclase (sGC),...

ATC Code: N06AX26 Wikipedia: Vortioxetine On September 30th 2013, FDA approved Vortioxetine (as the hydrobromide salt; tradename: Britellix ; research code: Lu AA21004 (Lu AA21004 (HBR) for the hydrobromide salt); ChEMBL: CHEMBL2104993 ), a multimodal antidepressant indicated for the treatment of major depressive disorder (MDD) . MDD is a mental disorder characterised by low mood and/or loss of pleasure in most activities, and by symptoms or signs such as increased fatigue, change in appetite or weight, insomnia or excessive sleeping and suicide attempts or thoughts of suicide. MDD is believed to arise from low levels of neurotransmitters (primarily serotonin (5-HT), norepinepherine (NE) and dopamine(DA)) in the synaptic cleft between neurons in the brain. Several antidepressants for the treatment of MDD are already available in the market and its choice depends on which symptoms need to be tackled. The most important classes of antidepressants include the Selective Serotonin ...

ATC Code: R03AK10 Wikipedia: Vilanterol On May 10 th , the FDA approved Vilanterol (Tradename: Breo Ellipta ; Research Code: GW-642444M), a long-acting beta 2 -adrenergic agonist , in combination with the already approved fluticasone furoate , an inhaled corticosteroid , for the long-term maintenance treatment of bronchospasm associated with chronic obstructive pulmonary disease (COPD). Chronic obstructive pulmonary disease (COPD) is characterised by the occurrence of chronic bronchitis or emphysema , a pair of commonly co-existing diseases of the lungs in which the airways become narrowed. Bronchial spasms, a sudden constriction of the muscles in the walls of the bronchioles , occur frequently in COPD. Vilanterol is a new long-acting beta 2  receptor agonist that through the activation of the beta 2 adrenergic receptors present in the bronchial smooth muscle, leads to bronchodilation, and consequently eases the symptoms of COPD. The beta 2 adrenergeic re...

ATC Code: V08CA02 Wikipedia: Gadoteric Acid On March 20th 2013, FDA approved Gadoteric Acid (as the meglumine salt; tradename: Dotarem ; research code: P 449; CHEMBL: CHEMBL2219415 ), a gadolinium-based contrast agent (GBCA) indicated for intravenous use with magnetic resonance imaging (MRI) in brain (intracranial), spine and associated tissues of patients ages 2 years and older, to detect and visualize areas with disruption of the blood brain barrier (BBB) and/or abnormal vascularity of the central nervous system (CNS). When placed in a magnetic field, Gadoteric Acid develops a magnetic moment. This magnetic moment enhances the relaxation rates of water protons in its vicinity, leading to an increase in signal intensity (brightness) of tissues. Gadoteric Acid enhances the contrast in MRI images, by shortening the spin-lattice (T1) and the spin-spin (T2) relaxation times. Other GBCAs have already been approved by FDA for use in patients undergoing CNS MRI and these inc...

ATC Code: Not Assigned Wikipedia: Ospemifene On February 26, FDA approved Ospemifene ( Trade Name : OSPHENA ,  PubChem :  CID 3036505 ,  ChEMBL :  CHEMBL210 5395 , ChemSpider :  2300501 ) for the treatment of moderate to severe Dyspareunia  - symptom of vulvular and vaginal atrophy due to menopause. Dyspareunia , is pain during or after sexual intercourse. It can affect men, but is significantly more common in women, affecting up to one-fifth of women at some point in their lives. Women with dyspareunia may have pain in the vagina, clitoris or labia. This may be due to medical or psychological causes. There are numerous medical causes of Dyspareunia, like : Vaginismus , Pelvic Inflammatory Disease , Genital or Pelvic Tumors, Urethritis , Urinary Tract Infection , Vaginal Atrophy , Vaginal Dryness , Vulvar Cancer , Childbirth Trauma (postpartum), Skin Conditions (Lichen Sclerosus, Lichen Planus, Eczema, Psoriasis), Female ...