Cystic Fibrosis Drugs in Development

Bronchitol, Denufosol, Vertex Drugs and Others Highlighted in Review

This month’s edition of Nature Reviews in Drug Discovery features a review of new treatments for cystic fibrosis that are in the process of undergoing clinical trials. The review is short, but it is exciting to see these drugs highlighted in a prestigious scientific publication like Nature. Some interesting content includes the future perspective on the therapies that will be most prevalent for treating CF over the next ten years, and a chart of the biggest drug development programs, shown below:

Some of these have been highlighted in previous posts (the Vertex drugs, Denufosol). Another drug featured in the review that seems quite promising is Bronchitol. This drug is an inhaled dry powder that encourages the flow of water across the lung surface in order to reduce stickiness of the mucus. The dry powder formulation offers several advantages: it can penetrate the lower airways more effectively than nebulized products (like Pulmozyme), and it is easy and convenient for patients to use as no nebulizer is necessary. The drug is currently in Phase III of testing, and the article predicts that it will have a large impact on future CF treatment if approved. The convenience of the dry powder means it could potentially reduce the burden of nebulized medications and inhaled antibiotics, and therefore has significant potential to positively affect quality of life for CF patients.

Some other drugs mentioned in the article include Moli1901, a drug designed for regulating salt transport (like Denufosol), and two antibiotics - Aztreonam and a dry powder formulation of tobramycin. Many of these drugs are anticipated to be available in 2009. More products available for treating CF means potential for better lung function and longer lives!

Reference: Storey S and Wald G. Novel agents in cystic fibrosis. Nature Reviews Drug Discovery: 2008 Jul;7(7):555-6.