Read-Through Agent (Premature Stop Codon Suppressor) Pregnancy: §4.6: as a precautionary measure it is recommended to avoid the use of ataluren during pregnancy; breast-feeding should be discontinued during treatment with ataluren.

Ataluren

Brand names: Translarna

An oral agent used to treat Duchenne muscular dystrophy caused by a nonsense (premature stop codon) mutation in ambulatory patients.

Auto-extracted from the source labelling — not yet independently clinician-verified. These values were distilled from the UK SPC (or the US label where noted) but have not had a clinician sign-off. Confirm against the current SmPC before prescribing.

Adult dose

Dose: 10 mg/kg body weight in the morning, 10 mg/kg body weight at midday and 20 mg/kg body weight in the evening (total daily dose 40 mg/kg body weight)

Route: oral (granules for oral suspension)

Frequency: three times daily — morning, midday and evening. Recommended dosing intervals: 6 hours between morning and midday doses, 6 hours between midday and evening doses, and 12 hours between the evening dose and the first dose the next day.

Max: 40 mg/kg/day is the recommended dose; the SPC states 'It is important to administer the correct dose. Increasing the dose above the recommended dose may be associated with reduced effectiveness.'

SPC §4.2 (Translarna 1000 mg granules for oral suspension). Treatment should only be initiated by specialist physicians with experience in the management of Duchenne/Becker muscular dystrophy. Translarna is available in sachets of 125 mg, 250 mg or 1000 mg; the SPC contains a body-weight-range table giving which sachet strength(s) to use — that table was not captured in this bundle and must be read from the SPC before dispensing. Delayed/missed dose: if the delay is less than 3 hours after the morning or midday dose, or less than 6 hours after the evening dose, take the dose with no change to subsequent scheduling; if the delay is longer, the dose should not be taken and the usual schedule resumed. Do not take a double or extra dose. Elderly: safety and efficacy in patients aged 65 and older have not yet been established. Hepatic impairment: no dosage adjustment required in mild, moderate or severe hepatic impairment. §4.4: patients must have a nonsense mutation in the dystrophin gene, as determined by genetic testing; monitor total cholesterol, LDL, HDL and triglycerides annually; monitor resting systolic and diastolic blood pressure every 6 months if given with systemic corticosteroids; monitor serum creatinine, BUN and cystatin C every 6 to 12 months. Method of administration: mix the full contents of each sachet with at least 30 ml of liquid (water, milk, fruit juice) or 3 tablespoons of semi-solid food (yoghurt or apple sauce), mix well, and take the entire dose; sachets should only be opened at the time of dose preparation.

Paediatric dose

Dose: 40 mg/kg

Route: oral (granules for oral suspension)

Frequency: Total 40 mg/kg/day given in 3 doses: 10 mg/kg in the morning, 10 mg/kg at midday and 20 mg/kg in the evening

Max: 40 mg/kg/day is the recommended dose; increasing the dose above the recommended dose may be associated with reduced effectiveness

SPC §4.2 Paediatric population: 'Paediatric patients with body weight ≥12 kg are treated as per the dosing recommendations by body weight range... The recommended dose is the same for all age ranges, i.e. 10 mg/kg body weight in the morning, 10 mg/kg body weight at midday, and 20 mg/kg body weight in the evening (for a total daily dose of 40 mg/kg body weight).' The safety and efficacy of Translarna in children <12 kg and aged 6 months to <2 years have not been established. Sachet strengths must be selected using the body-weight-range table in the SPC (not captured in this bundle). Verify against a children's formulary before prescribing.

Dose adjustments

Renal

No dosage adjustment is required for patients with mild or moderate renal impairment. Treatment of patients with severe renal impairment (eGFR <30 ml/min) or end-stage renal disease is not recommended; §4.4 adds that such patients should be treated only if the anticipated clinical benefit outweighs the potential risk, should be closely monitored, a lower ataluren dose should be considered, and treatment should not be initiated in previously untreated patients with eGFR <30 ml/min.

Dose auto-extracted from UK Summary of Product Characteristics (SPC) via the eMC — not yet clinician-verified. Always confirm against the product SmPC and your local formulary before prescribing.

Paediatric weight-based calculator

Patient weight (kg)

Verify in a children's formulary

Contraindications

Hypersensitivity to the active substance or to any of the excipients listed in section 6.1
Concomitant use of intravenous aminoglycosides

Side effects

Vomiting — the most common adverse reaction, occurring in ≥5% of all ataluren-treated patients
Upper abdominal pain (reported as a treatment-related serious adverse event, moderate in severity)
Constipation (led to treatment discontinuation in 1/416 patients)
Decreased appetite (very common/common per Table 1)
Hypertriglyceridaemia and changes in lipid profile (increased triglycerides and cholesterol)
In patients aged 2-5 years, a higher frequency of malaise, pyrexia, ear infection and rash was reported than in patients 5 years and older
NOTE: the §4.8 adverse-reaction table was truncated at the source-fetch limit — this list is incomplete

Interactions

Intravenous aminoglycosides — contraindicated; aminoglycosides reduce the readthrough activity of ataluren in vitro and ataluren was found to increase nephrotoxicity of intravenous aminoglycosides (§4.3/§4.4)
Other nephrotoxic medicinal products — concomitant use is not recommended; if unavoidable (e.g. vancomycin to treat MRSA) careful monitoring is required (§4.4)
Inducers of UGT1A9, and substrates of OAT1 or OAT3 — caution should be exercised when co-administered (§4.4)
NOTE: §4.5 was not retrieved in this bundle (the §4.4 text was truncated at the source-fetch limit) — check the full SPC for the complete interactions list

Clinical monograph

How it works

It promotes readthrough of premature stop codons during translation, allowing production of functional full-length dystrophin protein.

Prescribing in practice

It is only appropriate where a nonsense mutation in the dystrophin gene has been confirmed by genetic testing, and is not effective for other mutation types.
It should not be co-administered with intravenous aminoglycosides, which may increase the risk of nephrotoxicity, and renal function should be watched.
Treatment must be initiated and supervised by a specialist in the management of Duchenne muscular dystrophy.

Monitoring

Monitor renal function and lipids periodically and assess ambulatory function to gauge ongoing clinical benefit.

Counselling the patient

Take the medicine with food at the times instructed for steady absorption.
Attend appointments for blood tests checking kidney function.
Tell the team about all other medicines, especially any intravenous antibiotics.

Evidence & guidelines

Availability has been through managed access arrangements informed by clinical trials in nonsense-mutation Duchenne muscular dystrophy.

Reference: NICE TA420 (Ataluren for treating nmDMD, 2016 updated); EMA EPAR for Translarna; Parent Project Muscular Dystrophy guidelines; Confirm identity and dosing against the manufacturer SPC (eMC) and NICE. Verify against your local formulary and current prescribing references before prescribing. The structured dose values shown have been reviewed by a clinician. Monograph status: clinician-reviewed (2026-07-04).

Curated clinical cross-links plus same-class fallbacks.

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