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Cystic Fibrosis Market Snapshot: Disease-Modifying Drugs Elusive 24 Years After Discovery Of Root Cause

The discovery in 1989 of the underlying genetics causing cystic fibrosis was expected to spur broad development of disease-modifying therapies. As of 2013, however, only one such drug is on the market, for a small subset of CF patients, and a few other candidates are in clinical development.

Genomic research has increased scientific understanding significantly and opened up new pathways and targets for fighting a broad array of diseases. But while the underlying cause of cystic fibrosis – genetic mutations of the cystic fibrosis transmembrane conductance receptor protein (CFTR) – was discovered in 1989, development of disease-modifying agents against the disease based on that knowledge has been excruciatingly slow.

In 2012, Vertex Pharmaceuticals Inc

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