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Foundation receives $3.3-billion windfall for Kalydeco

Nature Biotechnology volume 33, pages 8–9 (2015)Cite this article

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A Correction to this article was published on 06 March 2015

This article has been updated

The payout represents an extraordinary return on the $150 million that Bethesda, Maryland–based CFF has invested in the programs since 2000. Worth 20 times the CFF's annual research budget to date, the payout has drawn attention to the growing role of disease foundations in funding commercial drug discovery and development. It has also inflamed the drug pricing debate, and raised questions about the levels of return appropriate for nonprofits and concerns over potentially tougher demands on academic scientists.

Still, as far as CFF and several other disease foundations are concerned, the payout represents a justified reward for a series of risky investments that could easily have failed. “We're willing to take risk,” says Robert Beall, president and CEO of CFF. “It took 13 years to get from [the initial investment in] Aurora [Biosciences] to Kalydeco,” he continues. Vertex's Kalydeco (ivacaftor) is the first disease-modifying therapy approved for a rare form of cystic fibrosis (Nat. Biotechnol. 30, 201–202, 2012). Beall claims Vertex—whose market capitalization is over $28 billion—wouldn't be there today “if not for CFF's investment. We take some return, sure, but it's for the patients,” he says. This argument is the central tenet of venture philanthropy: supporting commercial R&D programs to accelerate new treatments, but also to gain financial return, which is then ploughed back to help patients further.

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Change history

  • 23 February 2015

    In the version of this article initially published, Howard Fillit's affiliation was given as the Alzheimer's Disease Research Foundation instead of the Alzheimer's Drug Discovery Foundation. The error has been corrected in the HTML and PDF versions of the article.

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    Melanie Senior

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Senior, M. Foundation receives $3.3-billion windfall for Kalydeco. Nat Biotechnol 33, 8–9 (2015). https://doi.org/10.1038/nbt0115-8

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