Statement from the Cystic Fibrosis Foundation on Passage of the EXPERRT Act in the U.S. Senate

Statement from the Cystic Fibrosis Foundation on Passage of the EXPERRT Act in
                               the U.S. Senate

PR Newswire

BETHESDA, Md., June 27, 2012

Legislation Passes Last Hurdle Before Going to President for Signature

Bill Increases Patient and Expert Participation in FDA Review of Cystic
Fibrosis and Other Rare Disease Drugs

BETHESDA, Md., June 27, 2012 /PRNewswire-USNewswire/ --The Cystic Fibrosis
Foundation applauds passage of a bill in the United States Senate to expand
cooperation between the Food and Drug Administration (FDA) and outside rare
disease experts and patient advocates during the FDA drug approval process. 

The bill, passed yesterday, is known as the Expanding and Promoting Expertise
in Review of Rare Treatments Act (EXPERRT). Its passage in the Senate is the
last hurdle before the bill goes to President Obama for his signature. He is
expected to sign it into law.

Introduced by Sen. Sheldon Whitehouse (D-RI), EXPERRT is part of the FDA
Safety and Innovation Act, which reauthorizes the FDA's user-fee program that
funds its drug and device evaluation.

"The Cystic Fibrosis Foundation commends the Senate for its action on this
issue, which is critical to the 30,000 Americans with cystic fibrosis and
millions of others who struggle with complex conditions for which there are
little or no available treatments," said Robert J. Beall, Ph.D., president and
CEO of the Cystic Fibrosis Foundation. "Senator Whitehouse has been an
invaluable champion for the CF community, and we are grateful to him for his
work on this critical legislation."

"I've heard from so many people with rare diseases who are frustrated with
waiting for promising new treatments to gain approval, and who want to help
speed up the process," said Whitehouse. "This bill accomplishes that goal by
encouraging the FDA to take advantage of the wisdom and insights of rare
disease experts and patient advocates in approving new treatments. It's an
important step toward providing faster access to promising new therapies, and
I thank everyone who supported this bill for helping me to push it across the
finish line." 

Passage of this legislation follows the recent approval of Kalydeco™, a cystic
fibrosis drug developed by Vertex Pharmaceuticals with financial, scientific
and clinical support from the Cystic Fibrosis Foundation. Kalydeco's swift
approval, announced by the FDA just three months after it was submitted for
review, demonstrates how a well-informed, collaborative FDA review process
involving reviewers, industry partners, patients and cystic fibrosis experts
can efficiently move lifesaving therapies to market. Kalydeco is the first CF
drug to address the underlying cause of the disease in select CF patient

About the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world's leader in the search for a cure
for cystic fibrosis. The Foundation funds more cystic fibrosis research than
any other organization, and nearly every CF drug available today was made
possible because of Foundation support. Based in Bethesda, Md., the Foundation
also supports and accredits a national care center network that has been
recognized by the National Institutes of Health as a model of care for a
chronic disease. The Cystic Fibrosis Foundation is a donor-supported nonprofit
organization. For more information, go to

SOURCE Cystic Fibrosis Foundation

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