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).

Common side effects of ataluren include:

• Vomiting
• Diarrhea
• Nausea
• Headache
• Stomach upset or pain
• Gas (flatulence)
• Decreased appetite
• Weight loss
• High triglycerides
• High blood pressure (hypertension)
• Cough
• Nosebleeds
• Abdominal discomfort
• Constipation
• Rash
• Pain in the arms, legs, and chest muscles
• Blood in the urine
• Urinary incontinence
• Fever

This document does not contain all possible side effects and others may occur. Check with your physician for additional information about side effects.

If your doctor has directed you to use this medication, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, health care provider, or pharmacist first.

Interactions with ataluren include:

• aminoglycosides
• rifampicin
• oseltamivir
• acyclovir
• captopril
• furosemide
• bumetanide
• valsartan
• pravastatin
• rosuvastatin
• atorvastatin
• pitavastatin
• ciprofloxacin

Warnings

• This medication contains ataluren. Do not take Translarna if you are allergic to ataluren or any ingredients contained in this drug.

Contraindications

• Hypersensitivity to the active substance or any of the inactive ingredients: polydextrose, macrogol, poloxamer, mannitol, crospovidone, hydroxyethylcellulose, artificial vanilla flavor (maltodextrin, artificial flavors, and propylene glycol), silica - colloidal anhydrous, and magnesium stearate
• Concomitant use of intravenous aminoglycosides

Effects of Drug Abuse

• No information is available.

Short-Term Effects

• See "What Are Side Effects Associated with Using Ataluren?"

Long-Term Effects

• See "What Are Side Effects Associated with Using Ataluren?"

Cautions

• Caution should be exercised when ataluren is co-administered with medicinal products that are inducers of UGT1A9, or substrates of OAT1 or OAT3.

Pregnancy and Lactation

• There are no adequate data from the use of ataluren in pregnant women. Studies in animals have shown reproductive toxicity only at doses that resulted in maternal toxicity. Ataluren is not recommended for use during pregnancy.
• It is unknown whether ataluren is excreted in human milk. Available pharmacodynamic/toxicological data in animals have shown excretion of ataluren/metabolites in milk. Breastfeeding is not recommended during treatment with ataluren.