Ataluren

Generic name
Ataluren
Brand name
ATC Code
M09AX03
Dosages
Side effects in children
Warnings & precautions in children
Contra-indications in children

Interactions
PK
Renal impairment
References

Pharmacokinetics in children

F = 55%
T½: 2-6 hours

dose recommendation of formulary compared to licensed use (on-label versus off-label)

No information is present at this moment.

Available formulations

No information is present at this moment.

Dosages

Duchenne muscular dystrophy in outpatients
  • Oral
    • ≥ 2 years and ≥ 12 kg
      • 40 mg/kg/day divided in 3 uneven doses of 10-10-20 mg/kg/dosis.

      • Ataluren is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients. Efficacy has not been demonstrated in non-ambulatory patients. The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing.[1]

Renal impaiment in children > 3 months

  • GFR ≥30: dose adjustment not needed
  • GFR 10-30: general advice cannot be given; consider whether the intended effect of ataluren outweighs the potential risks
  • GFR <10: general advice cannot be given; consider whether intended effect of ataluren outweighs possible risks
Clinical consequences

With impaired renal function, exposure to ataluren and the acylglucuronide metabolite increases. This increases the risk of adverse reactions.

Clinical effects:
The toxicity of the metabolite is not known. In a study with 1-mal ataluren, the exposure ataluren in subjects with ClCr <30 ml/min was 61% higher compared to subjects with normal renal function. The exposure acylglucuronide metabolite was 3-8x higher.

The complete list of all undesirable drug reactions can be found in the national Summary of Product Characteristics (SmPC) – click here

Side effects in children

The side effect profile in children matches that of adults.

The complete list of all contra-indications can be found in the national Summary of Product Characteristics (SmPC) – click here

Contra-indications

No information available on specific contra indications in children.

The complete list of all warnings and precautions can be found in the national Summary of Product Characteristics (SmPC) – click here

Warnings & precautions

No information available on specific warnings and precautions in children.

Interactions

The complete list of all interactions can be found in the national Summary of Product Characteristics (SmPC) – click here

OTHER DRUGS FOR DISORDERS OF THE MUSCULO-SKELETAL SYSTEM

This pages provides a list of drugs from the same ATC class for comparison. This does not necessarily mean that these drugs are interchangeable.

Other drugs for disorders of the musculo-skeletal system
M09AX07
M09AX09
M09AX10
M09AX09

References

  1. PTC Therapeutics International Limited, SmPC Translarna (EU/1/13/902/003) 31-08-2018, www.geneesmiddeleninformatiebank
  2. NKFK Workinggroup Acute Kidney Impairment, Extrapolation of KNMP risk analysis "Impaired renal function" for adults to children, 20 Dec 2021

Changes

Therapeutic Drug Monitoring


Overdose