Translarna
Translarna Uses, Dosage, Side Effects, Food Interaction and all others data.
Translarna is a novel, orally administered drug that targets nonsense mutations. Translarna is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk. More specifically, ataluren is used in the small group of patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene.
This drug does not yet have approval by the US Food and Drug Administration or by Health Canada for any indications.
| Trade Name | Translarna |
| Generic | Ataluren |
| Ataluren Other Names | Ataluren |
| Type | oral suspension |
| Formula | C15H9FN2O3 |
| Weight | Average: 284.242 Monoisotopic: 284.059720369 |
| Protein binding | Ataluren is 99.6% bound to human plasma proteins and the binding is independent of plasma concentration. Ataluren does not distribute into red blood cells . |
| Groups | Approved, Investigational |
| Therapeutic Class | |
| Manufacturer | PTC Therapeutics Ltd |
| Available Country | United Kingdom, |
| Last Updated: | September 19, 2023 at 7:00 am |
Uses
Translarna is a medication used for the treatment of Duchenne muscular dystrophy.
Translarna is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk. More specifically, ataluren is used in the small group of patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene.
Translarna is also used to associated treatment for these conditions: Duchenne's Muscular Dystrophy (DMD)
How Translarna works
Translarna enables ribosomal readthrough of mRNA containing premature stop codons that otherwise would result in premature termination of protein chains. Use of ataluren allows cellular machinery to bypass nonsense mutations in genetic material, continue the translation process, and thereby restore the production of a full-length, functional protein.
The research on the effects of Translarna on the translation and stability of nonsense-containing mRNA in vitor show that Translarna 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, Translarna did not discriminate significantly between the UAG and UAA mRNAs. Translarna 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 Translarna modulates termination efficiency at premature nonsense codons.
Food Interaction
No interactions found.Elimination Route
Peak plasma levels of ataluren are attained approximately 1.5 hours after dosing in subjects who received medicinal product within 30 minutes of a meal .
Half Life
Translarna plasma half-life ranges from 2-6 hours and is unaffected either by dose or repeated administration .
Elimination Route
After a single oral dose of radiolabeled ataluren, approximately half of the administered radioactive dose is recovered in the faeces and the remainder was recovered in the urine. In the urine, unchanged ataluren and the acyl glucuronide metabolite account for less than 1% and 49%, respectively, of the administered dose .
Innovators Monograph
You find simplified version here Translarna
