The ChEMBL-og

ATC Code: A06AH03 Wikipedia:  Naloxegol ChEMBL:  CHEMBL2219418 On September 16th  FDA approved  Movantik (naloxegol, AZ-13337019 ), as an oral treatment for patients with opioid-induced constipation and chronic non-cancer pain. Naloxegol Naloxegol is an opioid receptor antagonist .  Due to its similarity to noroxymorphone, a main metabolite of oxycodone , naloxegol is classed as a controlled substance. However, the FDA analysed its abuse potential and concluded that there was no risk of dependency. Mode of Action Opioids are a class of drugs which are used to manage pain, but have a common side effect of reducing the motility of the gastrointestinal tract, making bowel movements difficult.  Opioids work by binding to the mu-receptors ( CHEMBL233 , UniProt:P35372 ) in the central nervous system, thereby reducing pain. However, they are also able to bind to the mu-receptors in the gastrointestinal tract, hence causing op...

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: Siltuximab ChEMBL: CHEMBL1743070 On April 23rd 2014 the FDA approved Siltuximab (Sylvant™) for the treatment of patients with multicentric Castleman’s disease (MCD) who are human immunodeficiency virus (HIV-)-negative and human herpes virus-8 (HHV-8)-negative. Castleman disease (Also known as giant or angiofollicular lymph node hyperplasia, lymphoid hamartoma, or angiofollicular lymph node hyperplasia) is an abnormal non-cancerous growth of the lymph node that can resemble lymphomas. It is contributed to by hyperproliferation of cytokine-producing lymphocytes. Castleman disease can be unicentric (involving a single lymph node) or multicentric (systemic). Siltuximab is approved for the multi centric disease. Overproduction of IL-6 has been linked to systemic manifestations in patients with MCD. Siltuximab is a chimeric (human and mouse) anti-IL6 antibody. It binds human IL-6 thus preventing the interaction of IL-6 to both soluble and membrane- bound ...

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: N05CH Wikipedia: Tasimelteon On January 31 st  2014, the FDA approved Tasimelteon (Tradename: Hetlioz ; Research Code(s): VEC-162, BMS-214778), a melatonin receptor agonist , for the treatment of Non-24-hour sleep-wake disorder (Non-24). Non-24-hour sleep–wake disorder (Non-24) is a chronic circadian rhythm sleep disorder, mostly affecting blind people. It is characterised by insomnia or excessive sleepiness related to abnormal synchronization between the 24-hour light–dark cycle and the endogenous circadian cycle (slightly longer than 24 hours). This deviation can be corrected by exposure to solar light, which resets the internal clock, however, the loss of photic input, and the absence of light perception in the majority of patients, prevents them from drifting back into normal alignment. Tasimelteon is an agonist at melatonin MT1 and MT2 receptors , with a relative greater affinity for MT2. These receptors are thought to be involved in the control...