The ChEMBL-og

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 (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: V09AX04 On October 25 th , the FDA approved Flutemetamol F18 (Tradename: Vizamyl ; Research Code: [ 18 F]AH110690 ), a radioactive diagnostic agent, for intravenous (i.v.) use in Positron Emission Tomography (PET) imaging of the brain in adult patients with cognitive impairment , who are being evaluated for Alzheimer’s disease (AD) and dementia. Alzheimer's disease is a non-treatable, progressively worsening and fatal disease, characterised by a decrease in cognitive functions, such as memory, and is usually associated with an accumulation of β amyloid (Uniprot: P05067 ) plaques in several brain regions. These deposits are believed to be responsible for cellular damage and ultimately cell death. Flutemetamol F18 is the second approved diagnostic drug to estimate β-amyloid neuritic plaque density, after the approval of Florbetapir F18 in 2012. Like Florbetapir F18, Flutemetamol F18 binds to β amyloid plaques in the brain where the F-18 isotope produces...

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: C10AX11 Wikipedia: Mipomersen On January 29 st , the FDA approved Mipomersen (Tradename: Kynamro ; Research Code: ISIS-310312), an oligonucleotide inhibitor of apolipoprotein B-100 (apo B-100) synthesis, indicated as an adjunct to lipid-lowering medications and diet to reduce low density lipoprotein-cholesterol (LDL-C) , apolipoprotein B (apo B), total cholesterol (TC) , and non-high density lipoprotein-cholesterol (non HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH) . Familial hypercholesterolemia is a genetic disorder, characterised by high levels of cholesterol rich low-density lipoproteins (LDL-C) in the blood. This genetic condition is generally attributed to a faulty mutation in the LDL receptor (LDLR) gene, which mediates the endocytosis of LDL-C. Mipomersen is the first antisense oligonucleotide that targets messenger RNA (mRNA) enconding apolipoprotein B-100 (Apo B-100), the principal apolipoprotein of LDL and its metabolic pre...

On October 17, the FDA approved Ocriplasmin (tradename: Jetrea ; Research Code: Microplasmin), a proteolytic enzyme indicated for the treatment of symptomatic vitreomacular adhesion (VMA) . VMA is a condition of the eye that results from the liquefaction of the vitreous gel within the human eye and consequent adhesion to the retina . As the eye ages, the vitreous humor can naturally separate from the retina. However, if the separation is not complete, areas of adhesion can occur. The traction from these adhesion areas on the retinal surface is the underlying pathology of symptomatic VMA, which can lead to ocular damage. Ocriplasmin is the first drug approved to treat this condition and it exherts its therapeutic action by selectively breaking down the three major protein components, fibronectin , laminin and collagen , of the vitreous body and vitreoretinal interface, and thereby dissolving the protein matrix responsible for VMA. The only alternative treatment is a surgica...

ATC code:  L01XX40 Wikipedia: Omacetaxine_mepesuccinate On October 22nd 2012 the FDA approved omacetaxine mepesuccinate (research code: CGX-635, trivial name: Homoharringtonine, trademark: Synribo TM ) for the treatment of chronic or accelerated phase chronic myeloid leukaemia ( CML ) in adults with resistance to two or more tyrosine kinase inhibitors. Omacetaxine is an old drug identified 35 years ago and known to have activity in CML, but its clinical development was previously halted due to the discovery of BCL-ABL and other targeted kinase inhibitors Pubmed: 21294709 . The rapid development of tyrosine kinase inhibitor resistant tumors has led to the need for agents that can act in these treatment-derived drug-resistant patients. Omacetaxine mepesuccinate has been approved based on observed major cytogenetic response rather than on improvement in disease-related symptoms or increased survival. Omacetaxine mepesuccinate/homoharr...

ATC code :   A16AB11 Wikipedia : Taliglucerase alfa   On May 1, the FDA approved taliglucerase alfa for the treatment of Type I Gaucher's disease. Gaucher's disease is the most common of the lysosomal storage diseases . It is a hereditary disease caused by a deficiency of the enzyme β- glucocerebrosidase (Uniprot: P04062 ), also called β-Glucosidase. Gaucher's disease is a rare genetic disease with an incidence of 1 in 50,000 births and is considered an orphan disease. Type I Gaucher's disease is about 100 times more common in people of Ashkenazi jewish descent compared a north American population. Symptoms of type I Gaucher's disease begin typically in early adulthood and include enlarged liver and grossly enlarged spleen, impaired bone structure, anemia and low platelet levels, leading to prolonged bleeding and easy bruising. If enzyme replacement therapy (ERT) is available, the prognosis for patients with type I Gaucher's disease is good. ...

ATC code: V03AF09 The first FDA new drug approval of 2012 is Glucarpidase, approved on Jan 17th 2012. Glucarpidase (tradename: Voraxaze ; formerly known as carboxypeptidase-G2 or CPG2) is a carboxypeptidase enzyme indicated for the treatment of toxic plasma methotrexate (MTX) concentrations ( > 1 umol/L) in patients with delayed MTX clearance due to impaired renal function. MTX (ChEMBL: CHEMBL426 ) is an antifolate drug and is one of the most widely used anticancer agents. Unlike other anticancer agents, MTX can be safely administrated over a wide dose range. However, during treatment with high doses of MTX, patients may develop renal dysfunction. Since MTX is primarily cleared by renal excretion, this will lead to toxic levels of MTX. Glucarpidase acts by converting MTX to its inactive metabolites 2,4-diamino-N 10 -methylpteroic acid (DAMPA) and gluta mic acid, providing thus an alternate route of elimination to renal excretion. Glucarpidase (Unipr...