Ataluren and ABPA

Question

Hello,
Firstly, I would like to know where is the third phase of the drug ataluren, and secondly whether this drug is effective against ABPA in CF patients, or only effective on congestion?

Answer

Hello,
The Phase 3 clinical study about the drug you mention (name of study: PTC 124) has not concluded a significant advantage in the patient group taking the study drug compared to the group taking placebo. A significant improvement in FEV was indeed not observed but only in the patients of the PTC 124 treated group who did not inhale antibiotics from the family of aminoglycosides (gentamicin, tobramycin). However, these antibiotics, which were not the subject of the study, are already known to improve FEV by a partial corrective effect of the cellular dysfunction in patients with class I mutations corresponding to the population of the study (see BW Ramsey’s publication).
The Phase 3 has not validated the efficiency hypothesis emitted following the Phase 2 study (cf. M. Konstan’s publication). It has not concluded an "improvement of medical service rendered" of PTC 124, which, accordingly, has not obtained its marketing authorization.
Otherwise, I have not found results of clinical studies demonstrating efficacy of PTC 124 on allergic bronchopulmonary aspergillosis (ABPA) in CF patients.
Hope that answers your question.
Cordially.
Dr. Gilles RAULT

Références :

Targeting Class 1 mutations: update on Ataluren as a promising treatment for non sense mutation CF.
Konstan M, Accurso F, De Boeck K, Kerem E, Rowe S, Sermet-Gaudelus I et al.
Pediatr Pulmonol 2012;47(S35):108-9.

Intermittent administration of inhaled tobramycin in patients with cystic fibrosis.
Ramsey BW, Pepe MS, Quan JM, Otto KL, Montgomery AB, Williams-Warren J et al.
Cystic Fibrosis Inhaled Tobramycin Study Group.
N Engl J Med. 1999;340(1):23-30.

15.03.2013