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Clinical management of CF » CFTR modulators » Ataluren (PTC124)

Ataluren (PTC124)

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Ataluren is a read-through agent designed to overcome premature termination codons (PTCs), or nonsense mutations, in the CFTR gene. In a recent phase III trial, ataluren did not improve lung function and did not decrease pulmonary exacerbations in the overall population of patients with CF and nonsense-mutation who received this treatment, but it might be beneficial for patients not taking chronic inhaled tobramycin.1 Therefore, a further trial is planned of patients not taking nebulised aminoglycosides, which may have been interacting with ataluren.

The mechanism of action is shown below.

  1. Kerem E et al. Lancet Respir Med 2014;2:539–47