Ataluren Phase 3 Data Encouraging for Cystic Fibrosis

Ataluren, formerly known as PTC124 appears to be a viable treatment for Cystic Fibrosis. This oral compound targets CFTR, the protein product and faulty gene believed to be the cause of Cystic Fibrosis. Ataluren also appears to be particularly effective for patients with Cystic Fibrosis (CF) nonsense mutation. This genetic mutation interrupts the CFTR protein production, leading to shortened and abnormal functioning.

nonsense mutation

Encouraging data from a recently released Ataluren Phase 3 trial has offered hope to pharmacists, practitioners and patients with nonsense mutation CF, as revealed in Lancet Respiratory Medicine. Ataluren Phase 3 clinical trial results have shown positive trends in lung function from the participants who received Ataluren as compared to the placebo group. The patients who were administered with Ataluren had less pulmonary exacerbations, as well as stabilization in their Forced Expiratory Volume in 1 Second (FEV1) test results. The apparent stabilization of this rare nonsense mutation CF is especially important for this patient population, as this form of Cystic Fibrosis is more severe and in need of disease stabilization.Ataluren CAS 775304-57-9


The Ataluren Phase 3 study of Ataluren involved 232 patients in eleven countries. As a double-blind study 116 patients were given Ataluren and 116 patients were dosed with a placebo. The primary endpoint of the study was to demonstrate a change from baseline in the percent predicted FEV1 at 48 weeks . Results were encouraging, showing a significant positive trend in the FEV1 results from the patients who were administered Ataluren versus the trial group who were given a placebo. Additionally, there were 23 percent fewer pulmonary exacerbations documented in the group who received Ataluren, proving further benefits of this investigational compound. The tolerability and safety of Ataluren was evident, with the incidence of adverse events throughout the 48 week study being similar in both the Ataluren and placebo groups. An elevation in creatinine levels was noted in the patient population who were administered Ataluren along with adjunctive treatment with systemic aminoglycosides. There were reports of only mild to moderate adverse effects, none of which were life-threatening. Continued data is being gleaned from current trials of Ataluren, with the hope that this efficacious treatment will become a first-in-class treatment for patients with nonsense mutation Cystic Fibrosis.

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