R&D Trends: Cystic Fibrosis – New drugs tackle both cause and effect

Introduction

Two decades after the identification of the cystic fibrosis transmembrane conductance regulator protein, approved drugs capable of correcting dysfunctions in this protein are nearing reality. This report captures the thoughts of cystic fibrosis specialists on this exciting development, and also highlights potentially life-extending innovations among inhaled antibiotics.

Features and benefits

• Features a multi-parameter assessment of the cystic fibrosis pipeline from preclinical to preregistration.
• Identifies the most promising innovative new candidates within the pipeline.
• Analyzes the changes to the cystic fibrosis research portfolio over the past 2.5 years.
• Discusses the key learnings from candidates recently discontinued from late-stage cystic fibrosis clinical trials.
• In-depth interviews with four key opinion leaders reveal strong views on unmet needs and target product profiles.

Highlights

There remain significant unmet needs among cystic fibrosis (CF) patients, but commitment to the discovery of new therapies is growing. The pharmaceutical industry and CF organizations have increased their efforts significantly. Over the past 2.5 years the CF pipeline has grown by 11%, and the number of clinical trials active/recruiting has doubled.

Key opinion leaders interviewed by Datamonitor were unanimous in their praise for the great strides made by protein repair treatments. Courtesy of work done by Cystic Fibrosis Foundation Therapeutics, PTC Therapeutics and Vertex, there is now precedent for small molecule-mediated protein repair across the most common three classes of CF mutation.

An arms race is emerging between increasingly resilient lung bacterial infections and the next generation of inhaled antibiotics. CF specialists are looking to powerful broad-spectrum agents and/or biofilm-penetrating liposomal formulations to counter the threat posed by the rising incidence of aminoglycoside-resistant pathogens.

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