Ataluren

• 3 - [ 5 - ( 2-fluoro- phenyl) - [1,2,4] oxadiazol- 3-yl] -benzoic acid (IUPAC)
• PTC -124

Template: Infobox chemical / molecular formula search available

Ataluren is an experimental agent that is intended to provide for diseases of genetic origin, which are based on non-sense mutations, correct more of the gene on the stop codon addition.

The substance is a development of the company PTC Therapeutics, South Plainfield (New Jersey, USA). It is as a drug against cystic fibrosis ( CF) and Duchenne muscular dystrophy caught the eye and can be administered orally.

For both indications, the substance is registered by the European Commission as an orphan drug in the EU Community Register.

As a possible mechanism of action is stated that the lack of expression in cystic fibrosis of the CFTR protein restored by ataluren and thus the morbidly limited water and chloride ion transport is normalized from epithelial cells again. This is accompanied by the liquefaction of the cystic fibrosis pathologically viscous body secretions. A similar mechanism of action in this indication has also the antibiotic gentamicin, compared with this is to ataluren but fewer adverse effects and may (instead of gentamicin inhalation) be administered orally.

In animal experiments ataluren suggested in the muscle cells of mice to the formation of the protein dystrophin, the absence of which is held responsible for the muscle weakness. Therefore, the substance should also be developed for the treatment of Duchenne muscular dystrophy.

The plasma half-life of ataluren is 3 -6h.