ataluren

• Ataluren is an investigational treatment being developed to treat Duchenne muscular dystrophy (DMD) resulting from a nonsense mutation in the dystrophin gene in ambulatory patients aged 2 years and older, and Becker muscular dystrophy (BMD).
• The U.S Food and Drug Administration (FDA) accepted a new drug application for ataluren (Translarna) on March 6, 2017. On September 28, 2017, the FDA’s Peripheral and Central Nervous Systems Drugs Advisory Committee concluded the evidence to support the effectiveness of Translarna as a DMD treatment was inconclusive, and the application needed further research. On October 24, 2017, the FDA gave its final decision stating it is unable to approve the application due to the lack of substantial evidence of Translarna’s effectiveness and needs at least an additional adequate and well-controlled clinical trial to demonstrate the treatment’s effectiveness.
• The European Medicines Agency has authorized ataluren for use in the European Union (EU).
• Ataluren is available under the following different brand names: Translarna.

Dosages of Ataluren:

• Granules (125, 250 and 1,000 mg)

Dosage Considerations – Should be Given as Follows:

• Granules are to be taken by mouth after mixing them with liquid or semi-solid food (such as yogurt).
• Ataluren is taken three times a day, and the recommended dose is 10 mg/kg (10 mg per kilogram body weight) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total daily dose of 40 mg/kg).