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2018 Price of Ataluren
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Ataluren volume of distribution
Discount Cards 16,000+
Clients Benefit 29%
Total savings $4,735,080
What is Ataluren
Ataluren is a novel, orally administered drug that targets nonsense mutations. It is used as a treatment for Duchenne muscular dystrophy (DMD) and cystic fibrosis (CF), and has the potential to treat a number of other genetic disorders caused by nonsense mutations. On 23 May 2014 ataluren received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). Translarna was first available in Germany, the first EU country to launch the new medicine. In August 2014, ataluren received market authorization from the European Commission to treat patients with nonsense mutation Duchenne muscular dystrophy. A confirmatory phase III clinical trial is ongoing. The drug does not yet have approval by the US Food and Drug Administration.
Ataluren mechanism of action
Ataluren allowed the cellular machinery to bypass the nonsense mutation, continue the translation process, and thereby restore the production of a full-length, functional protein. The research on the effects of Ataluren on the translation and stability of nonsense-containing mRNA in vitor show that Ataluren promoted readthrough at each of the nonsense codons, showing maximal activity with UGA, while having no effect on mRNA levels. Unlike the stable cell line assays, Ataluren did not discriminate significantly between the UAG and UAA mRNAs. Ataluren was a more potent nonsense-suppressing agent than gentamicin, and exhibited 4- to 15-fold stimulation of in vitro readthrough relative to the controls at levels similar to those in the stable cell reporter assays. These results indicate that Ataluren modulates termination efficiency at premature nonsense codons.
Dosage forms of Ataluren
|Granules for oral suspension||Oral use||1000 mg|
|Granules for oral suspension||Oral use||125 mg|
|Granules for oral suspension||Oral use||250 mg|
Humans and other mammals
Indication of Ataluren
For the treatment of cystic fibrosis and muscular dystrophy.
Toxicity of Ataluren
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