A European Regulatory Perspective on Cystic Fibrosis: Current Treatments, Trends in Drug Development and Translational Challenges for CFTR Modulators

Overview

Journal Eur Respir Rev

Specialty Pulmonary Medicine

Date 2018 Apr 15

PMID 29653946

Citations 13

Authors

Stefano Ponzano

Giulia Nigrelli

Laura Fregonese

Irmgard Eichler

Fabio Bertozzi

Tiziano Bandiera

Luis J V Galietta

Marisa Papaluca

Affiliations

Soon will be listed here.

Abstract

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 ( 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.

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