The CF Pipeline

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Cystic Fibrosis is an orphan disease with approximately 30,000 individuals suffering from CF in the United States, and another 60,000 in the rest of the world. It is believed that CF affects an average of 1 in every 4000 individuals, with the rate varying based on ethnicity. Two new options in the pipeline offer patients a more upstream therapeutic approach.

• Inspire Pharmaceuticals - Denufosol tetrasodium

• PTC Therapeutics - PTC124 Session Highlights:

• Still undetermined where new treatments will fit into the current line of therapy.

Clinicians would like to treat patients with drugs that get at the major causes of disease, after newborns have been screened (bottom-up approach). However, both treatments have not been tested in children 5 and under, making it very difficult to use a “bottom-up approach. • More efficient and effective inhalation drug delivery will go a long way to decreasing the burden on patients. • Effectively finding treatments which aid patients through exacerbation events will help to improve patient lung function. These infections typical require patients to receive treatment in the hospital for 7-12 days with lung function not returning to pre-infection levels. Longer clinical trials are planned for both Denofosol and PTC124 to address this issue.