ataluren at a glance
Medicine class
A mutation-specific protein-restoration treatment.
Who it is for
Ambulatory people aged 2 years and older with genetically confirmed nonsense-mutation Duchenne muscular dystrophy.
Administration
Granules are mixed with liquid or soft food and taken as a suspension.
Prescribing
Treatment is initiated by a specialist experienced in Duchenne or Becker muscular dystrophy.
Safety monitoring
The product carries a black triangle for additional monitoring of suspected side effects.
What is ataluren used for?
Ataluren is licensed for:
Duchenne muscular dystrophy caused by a nonsense mutation in the dystrophin gene, in ambulatory people aged 2 years and older. Genetic testing must confirm the mutation.
How does ataluren work?
A nonsense mutation creates an early stop signal while dystrophin is being made, resulting in an incomplete protein. Ataluren helps the ribosome read through this signal so full-length dystrophin may be produced.
ataluren preparations and strengths
Oral suspension
Route: Oral
Strengths: 125 mg, 250 mg, 1000 mg
How to take ataluren
Use the sachets exactly as supplied and consume the whole prepared amount.
Open sachets only when preparing the medicine.
Mix the complete contents with at least 30 mL of water, milk or fruit juice, or 3 tablespoons of yoghurt or apple sauce.
Mix thoroughly and consume the entire preparation.
Use immediately where possible. Otherwise, follow the product storage limits.
ataluren side effects
Common or expected effects
- Vomiting
- Nausea, abdominal discomfort, upper abdominal pain, wind or constipation
- Reduced appetite or weight
- Headache
- Cough or nosebleed
- Raised blood pressure
- Rash
- Raised triglycerides or cholesterol
- Changes in kidney-function tests or blood in the urine
- Fever
Get urgent medical advice
- Severe allergic reaction, including breathing difficulty or swelling of the tongue or throat
- Severe or persistent vomiting, particularly if fluids cannot be kept down
- Markedly reduced urine or visible blood in the urine
ataluren in pregnancy
Human pregnancy data are inadequate. UK product information recommends avoiding ataluren during pregnancy as a precaution. Contact the specialist prescriber if pregnant, planning pregnancy or pregnancy occurs.
ataluren while breastfeeding
It is unknown whether ataluren enters human breast milk, and a risk to the infant cannot be excluded. UK product information states that breastfeeding should stop during treatment; discuss individual circumstances with the specialist team.
ataluren interactions
Tell the specialist and pharmacist about all prescribed, non-prescription and hospital-administered treatments. Important examples include:
Intravenous aminoglycoside antibiotics
Must not be given with ataluren because the combination may increase kidney toxicity and reduce ataluren's readthrough activity.
Other potentially kidney-toxic medicines, including vancomycin
Concurrent use is not recommended where avoidable. Kidney function requires careful monitoring if combined treatment is necessary.
Rifampicin and other UGT1A9 inducers
May reduce ataluren exposure and potentially affect its activity.
Common questions about ataluren
Answers are fully visible for fast scanning and source review.
Does ataluren work for every dystrophin-gene mutation?
No. Its action is specific to premature stop codons, so patients without a nonsense mutation should not receive it.
Is genetic testing required?
Yes. A nonsense mutation in the dystrophin gene must be confirmed.
Does ataluren cure Duchenne muscular dystrophy?
It is not a cure. It aims to increase production of full-length dystrophin, while multidisciplinary Duchenne care remains necessary.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.