RT Journal Article
SR Electronic
T1 A European regulatory perspective on cystic fibrosis: current treatments, trends in drug development and translational challenges for CFTR modulators
JF European Respiratory Review
JO EUROPEAN RESPIRATORY REVIEW
FD European Respiratory Society
SP 170124
DO 10.1183/16000617.0124-2017
VO 27
IS 148
A1 Stefano Ponzano
A1 Giulia Nigrelli
A1 Laura Fregonese
A1 Irmgard Eichler
A1 Fabio Bertozzi
A1 Tiziano Bandiera
A1 Luis J.V. Galietta
A1 Marisa Papaluca
YR 2018
UL http://err.ersjournals.com/content/27/148/170124.abstract
AB In this article we analyse the current authorised treatments and trends in early drug development for cystic fibrosis (CF) in the European Union for the time period 2000–2016. The analysis indicates a significant improvement in the innovation and development of new potential medicines for CF, shifting from products that act on the symptoms of the disease towards new therapies targeting the cause of CF. However, within these new innovative medicines, results for CF transmembrane conductance regulator (CFTR) modulators indicate that one major challenge for turning a CF concept product into an actual medicine for the benefit of patients resides in the fact that, although pre-clinical models have shown good predictability for certain mutations, a good correlation to clinical end-points or biomarkers (e.g. forced expiratory volume in 1 s and sweat chloride) for all mutations has not yet been achieved. In this respect, the use of alternative end-points and innovative nonclinical models could be helpful for the understanding of those translational discrepancies. Collaborative endeavours to promote further research and development in these areas as well as early dialogue with the regulatory bodies available at the European competent authorities are recommended.Alternative nonclinical models for CFTR modulators are needed to increase prediction of clinical efficacy http://ow.ly/Tj8y30iQNiD