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Meet the 2 Newest Investments in Cystic Fibrosis

By Maxx Chatsko - Sep 29, 2013 at 12:00PM

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Investors can now invest in two new companies eyeing the orphan disease. Are they worth it?

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 -2.50%) 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 -1.22%) 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.

Fool contributor Maxx Chatskohas no position in any stocks mentioned. Check out his personal portfolio or his CAPS page, or follow him on Twitter, @BlacknGoldFool, to keep up with his writing on biopharmaceuticals, industrial biotech, and the bioeconomy.

The Motley Fool recommends Vertex Pharmaceuticals. Try any of our Foolish newsletter services free for 30 days. We Fools don't all hold the same opinions, but we all believe that considering a diverse range of insights makes us better investors. The Motley Fool has a disclosure policy.

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Stocks Mentioned

Vertex Pharmaceuticals Incorporated Stock Quote
Vertex Pharmaceuticals Incorporated
VRTX
$292.16 (-1.22%) $-3.61
AbbVie Inc. Stock Quote
AbbVie Inc.
ABBV
$142.08 (0.81%) $1.14
PTC Therapeutics, Inc. Stock Quote
PTC Therapeutics, Inc.
PTCT
$52.01 (-3.16%) $-1.70
Roche Holding AG Stock Quote
Roche Holding AG
RHHBY
$41.04 (-2.50%) $-1.05

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