ataluren: prescribing and clinical use
Focused clinical detail for use alongside current product information, local policy and patient-specific assessment.
ataluren: clinical details
Prescribing considerations
- Confirm a nonsense mutation in the dystrophin gene by genetic testing before treatment.
- Licensed for ambulatory patients aged 2 years and older; initiation should be by an experienced specialist.
- Maintain adequate hydration, particularly during intercurrent illness.
- Review suitability if ambulation status or renal function changes.
- The authorisation is conditional and the product remains under additional monitoring.
Contraindications and cautions
- Hypersensitivity to ataluren or an excipient.
- Concurrent intravenous aminoglycoside treatment.
- Do not use without a confirmed nonsense mutation.
- Do not initiate in previously untreated patients with severe renal impairment; end-stage renal disease and severe impairment require specialist benefit–risk assessment.
Monitoring
- Monitor total cholesterol, LDL, HDL and triglycerides at least annually, or more often when clinically indicated.
- Monitor resting blood pressure every 6 months when systemic corticosteroids are also used.
- Monitor serum creatinine, blood urea nitrogen and cystatin C every 6–12 months, with closer surveillance where clinically indicated.
- Review nephrotoxic medicines and OAT1, OAT3, OATP1B3 or UGT1A9 interactions.
Clinical pharmacology
Ataluren promotes ribosomal readthrough of premature stop codons in messenger RNA. It is highly protein-bound, undergoes predominantly UGT1A9-mediated glucuronidation, and is eliminated through renal and hepatic excretion of its glucuronide metabolite.
Formulation and product differences
- White to off-white granules for oral suspension supplied in sachets.
- Available sachet strengths use the same preparation method and may be combined as prescribed.
- Prepared suspension is preferably used immediately; refrigerated or room-temperature preparations have limited in-use stability.
ataluren preparations and strengths
Oral suspension
Route: Oral
Strengths: 125 mg, 250 mg, 1000 mg
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.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.