Atalures

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Structural formula
Structure of PTC-124
General
Non-proprietary name Atalures
other names
  • 3- [5- (2-Fluoro-phenyl) - [1,2,4] oxadiazol-3-yl] -benzoic acid ( IUPAC )
  • PTC-124
Molecular formula C 15 H 9 FN 2 O 3
External identifiers / databases
CAS number 775304-57-9
EC number 922-364-8
ECHA InfoCard 100.132.097
PubChem 11219835
ChemSpider 9394889
DrugBank DB05016
Wikidata Q753330
Drug information
ATC code

M09 AX03

properties
Molar mass 284.24 g · mol -1
safety instructions
Please note the exemption from the labeling requirement for drugs, medical devices, cosmetics, food and animal feed
GHS hazard labeling
no classification available
As far as possible and customary, SI units are used. Unless otherwise noted, the data given apply to standard conditions .

Ataluren is a drug for the treatment of diseases of genetic origin, on nonsense - mutations that a reading of the based gene from the stop codon to give out.

Ataluren is approved under the name Translarna for the treatment of Duchenne muscular dystrophy . The substance is effective orally . Ataluren has also been considered as a drug for cystic fibrosis (cystic fibrosis).

Mechanism of action

In animal experiments, Ataluren stimulated the formation of the protein dystrophin in the muscle cells of mice , the lack of which is held responsible for the muscle weakness that occurs in Duchenne muscular dystrophy.

For the potential use in cystic fibrosis (cystic fibrosis), a possible mechanism of action is stated that ataluren restores the CFTR protein expression that is missing in this disease and thus normalizes the pathologically restricted transport of water and chloride ions from epithelial cells. This is accompanied by the liquefaction of the body secretions, which are pathologically viscous in cystic fibrosis. The antibiotic gentamicin has a comparable mechanism of action in this indication, but ataluren is said to have fewer undesirable effects compared to this and can also be administered orally (instead of inhalatively as gentamicin).

The plasma half-life of ataluren is 3 to 6 hours.

Development and approval

For the treatment of Duchenne muscular dystrophy and cystic fibrosis, the substance was registered by the European Commission in 2005 as an orphan drug in the EU community register.

In 2014, Ataluren was approved as Translarna (manufacturer: PTC Therapeutics , South Plainfield, New Jersey, USA) in the EU in the indication Duchenne muscular dystrophy in ambulatory patients from the age of 5 years. In May 2018, the approval for children aged 2 and over followed.

Other potential areas of application are the rare diseases Muscular Dystrophy Becker and Aniridia , for which Ataluren has also been granted orphan status.

Individual evidence

  1. ↑ This substance has either not yet been classified with regard to its hazardousness or a reliable and citable source has not yet been found.
  2. ↑ Summary of the EPAR for the public , EPAR of the EMA, accessed on June 13, 2018
  3. ↑ EMEA / COMP / 122996/2005 Rev.2 ( Memento of December 26, 2009 in the Internet Archive ) (PDF; 121 kB) Assessment of the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency, July 3, 2007.
  4. ↑ EM Welch, ER Barton et al. a .: PTC124 targets genetic disorders caused by nonsense mutations. In: Nature . Volume 447, number 7140, May 2007, pp. 87-91, doi: 10.1038 / nature05756 , PMID 17450125 .
  5. ↑ EMEA / COMP / 123054/2005 Rev.3 ( Memento of December 26, 2009 in the Internet Archive ) (PDF; 127 kB), assessment of the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency , July 3, 2007.
  6. ↑ S. Hirawat, EM Welch et al. a .: Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers. In: Journal of clinical pharmacology. Volume 47, Number 4, April 2007, pp. 430-444, doi: 10.1177 / 0091270006297140 , PMID 17389552 .
  7. ↑ Entry EU / 3/05/277 in the EU Community Register for Orphan Drugs. Retrieved February 28, 2019 .
  8. ↑ Entry EU / 3/05/278 in the EU Community Register for Orphan Drugs. Retrieved February 28, 2019 .
  9. ↑ Entry EU / 3/12/1010 in the EU Community Register for Orphan Drugs, accessed on February 28, 2019.
  10. ↑ Entry EU / 3/15/1561 in the EU Community Register for Orphan Drugs, accessed on February 28, 2019.

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