ATC code: A16AA05
On March 18th, the FDA approved carglumic acid to be marketed under the name Carbaglu as the first specific treatment for a rare form of hyperammonaemia. Carbaglu has been marketed in Europe since 2003. The ATC code is A16AA05.
Carglumic acid is used to treat a deficiency in the enzyme N-Acetyl Glutamate Synthase (NAGS), which leads to acute hyperammonaemia. NAGS deficiency is a very rare genetic disorder where a single base deletion in chromosome 17q leads to a frameshift mutation in the NAGS gene (UniProt: Q8N159) (this leads to a non-functional protein product). It is estimated that it occurs in one of ~50,000 births and due to the rareness of this autosomal recessive disorder, Carbaglu is considered as an orphan drug. NAGS deficiency manifests within the first week of life with unspecific symptoms such as failure to feed, low body temperature and sleepiness. If left untreated, the accumulation of ammonia (hyperammonemia) in the patient's blood causes letharg…
This update contains activity data from more than 1,000 additional publications, bringing the total number of distinct compounds to 530,790 (corresponding to 640,809 compounds records) and the total number of targets to 7,330. In addition, we have carried out widespread curation of the existing chemical structures in the database. Download versions of the new database will be available on our FTP site shortly.
ATC code: A16AB10
On 26th February, FDA has approved Velaglucerase Alfa, under the trade name VPRIV. Velaglucerase Alfa, is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease. Gaucher's disease is an autosomal recessive disorder characterized by a deficiency of the lysosomal enzyme beta-glucocerebrosidase (UniProt: P04062) (E.C. 18.104.22.168) (this enzyme is also known as glucosylceramidase, β-glucosidase, and D-glucosyl-N-acylsphingosine glucohydrolase).
Velaglucerase Alfa ATC code is A16AB10.
Glucocerebrosidase catalyses the hydrolysis of the glycolipid glucocerebroside, which is an intermediate in the glycolipid metabolism. Deficiency in this enzyme causes accumulation in the liver, spleen, bones, bone marrow and nervous system and can prevent cells and organs from working properly. Velaglucerase Alfa works by catalysing the hydrolysis of glucocerebroside, reducing the amoun…
New data and a slightly remodelled interface for Kinase SARfari are now available here.
Key changes are: Loaded chembl_02 data - Compound count increased from 17,028 to 28,223 and Bioactivity count increased from 68,861 to 146,865 Compound selected sets updated - 10 Drugs compounds, previously 7, and 258 Clinical Candidates, previously 53 Interface updated with EMBL-EBI standard styling Added Download section to interface
An interesting and thought provoking paper from last year was 'Heteroaromatic Rings of the Future' by Will Pitt (of UCB) (subscription required) and others at UCB. The basic idea of the paper was to exhaustively identify then analyse the class of all possible heterocycles with the following constraints. i) mono and bicyclic rings, ii) Only 5 and 6 membered rings, iii) Only containing C, N, O, S and H, iv) neutral, v) obey Hückel’s 4n+2 rule of aromaticity , and vi) Only exocyclic carbonyls. Heterocycles like this are at the very core of drug discovery and medicinal chemistry. The dataset is now available for download from the chembl ftp site, and also as a Google document The file contains... regid: the id for each distinct ring system SMILES: the encoded chemical structure of each ring system Training dataset hits: the count of substructure hits found in the
original search of commercial compound catalogues, drugs etc. (as reported in the paper). Beilstein hits: the count of substr…
ChEMBL-og readers may be interested in a position at the world-leading Institute of Cancer Research in Sutton. It is for a 'Higher Scientific Officer', with the job involving primarily programming and bio/chemical data integration, the post is within the Computational Biology and Chemogenomics team.
For more information see the ICR web site.