Individuals with cystic fibrosis, or CF, have relatively few treatment options available to them. Approved medicines largely focus on reducing inflammation and keeping infections at bay -- treating symptoms of the disease, not the cause. For instance, Pulmozyme from Roche (RHHBY -2.24%) was the first approved treatment for CF. The drug is an enzyme that breaks down mucus in the lungs, which improves breathing and the body's ability to clear such mucus. It fetched $305 million in sales for Roche in the first half of this year, but it is approved for multiple indications worldwide and is hardly the best the industry has to offer.
Promising new research in the genetics of CF is about to change that. Kalydeco from Vertex Pharmaceuticals (VRTX -1.02%) is approved to treat patients with a certain mutation, G551D, in the gene encoding the CFTR protein, which controls fluid flow in and out of cells. The drug has greatly improved the lives of cystic fibrosis patients with the mutation -- about 4% of all patients -- and generated sales of $160 million in the first half of 2013. Not very promising, but consider that pipeline candidates currently being evaluated in clinical trials that treat another genetic mutation, F508del, could allow Vertex to treat 90% of the cystic fibrosis population and generate peak sales of $1.7 billion by 2018.
The prospects have spurred others to dig into genetic data to unveil targets for treating cystic fibrosis more effectively. Who are the latest entrants? Are they investment-worthy on the backs of CF treatments alone? Fool contributor Maxx Chatsko explains in the following video.
