March 2012

FDA Approval of Kalydeco Opens Doors to New Research Efforts

“Kalydeco has the potential to transform the lives of people with the G551D mutation of CF, and represents hope for all people with the disease,” says Robert J. Beall, Ph.D., president and CEO of the Foundation.
“Kalydeco has the potential to
transform the lives of people with the G551D mutation of CF, and represents hope for all people with the disease,”
says Robert J. Beall, Ph.D., president
and CEO of the Foundation.

The Cystic Fibrosis Foundation kicked off the new year with the news that the U.S. Food and Drug Administration (FDA) had approved Kalydeco™ — the first drug to target the underlying cause of cystic fibrosis.

Kalydeco (formerly known as VX-770) was developed by Vertex Pharmaceuticals Inc. with significant support from the CF Foundation in a collaboration that began more than a decade ago. The FDA approved Kalydeco on Jan. 31, 2012, for people with the G551D mutation of CF ages 6 and older.

Kalydeco is now being tested in people with other CF mutations similar to G511D that hopefully will respond to Kalydeco when taken alone. The drug is also being tested in combination with another potential therapy, VX-809, in people with at least one copy of the most common mutation of CF, Delta F508.

Connections spoke with Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation, about what Kalydeco’s approval means for ongoing efforts to speed the development of drugs that treat the underlying cause of CF in all people living with the disease.

Why is the FDA approval of Kalydeco so significant?

This news is a significant breakthrough for the CF community. The approval of Kalydeco shows we know how to successfully treat the basic genetic defect in CF.

When we began this collaboration with Vertex, we didn’t know whether it was possible to treat the underlying cause of CF with an oral drug. It was a big risk, and people thought it wasn’t possible. But that didn’t stop us — we love doing the impossible.

The approval of Kalydeco shows that we can do it and that our drug development strategy has been right on the mark. Kalydeco has the potential to transform the lives of people with the G551D mutation of CF, and represents hope for all people with the disease.

What is being done to find a treatment for people with the most common mutation of CF?

Kalydeco has so far been shown to be effective for those with the G551D mutation, but the research behind the drug has opened exciting new doors that may one day lead to a cure for all people living with CF.

Later this year we expect to have results from the second part of the Phase 2 trial of Kalydeco and VX-809 in those with the Delta F508 mutation. This is a very important trial; nearly 90 percent of people with CF have the Delta F508 mutation.

Results from the first part of this Phase 2 trial showed that those who received the two drugs together had a decrease in sweat chloride levels — a sign that the combination treatment is having an effect on the root cause of the disease. 

Vertex is also testing Kalydeco together with another potential drug, VX-661, in people the most common mutation. These studies could lay the groundwork for future clinical research using a combination therapy approach.

What other developments can we expect in 2012?

This year we also expect to have results from another pivotal study — a large, international Phase 3 trial of ataluren, which is designed to treat the basic defect in CF in people who have what are known as “nonsense mutations.”

About 10 percent of people with CF have these mutations, which interrupt production of the CFTR protein — the key protein associated with CF — causing it to be too short and not function.

In an earlier trial of ataluren, those who received the drug showed improved CFTR function and the drug was also associated with an improvement in lung function. 

So by the end of 2012, we should know much more about treating the underlying cause of CF in greater numbers of people with the disease.

What is the next step in the Foundation’s drug development efforts?

Even with the significant progress that has been made with Kalydeco, we are not at the finish line. Developing new drugs is risky and expensive, and only one in five drugs that are in clinical trials ever gets approved for patients.

Our top priority now is accelerating the development of an effective treatment for people with the Delta F508 mutation.

We have significantly expanded our collaborations with other leading pharmaceutical companies, including Pfizer and Genzyme, to identify new potential drugs that target the underlying cause of CF. These new programs will use a variety of screening processes and draw on vast libraries of up to 6 million chemical compounds.

This is a very promising step forward in CF research, and will help speed the discovery of promising therapeutic candidates that can then be moved more quickly into clinical trials and potentially into the hands of people with CF.

Is there anything else you’d like to share?

The science behind Kalydeco has given us a roadmap to research and development that could eventually lead to a cure for all people with CF. We will not stop until we have reached this goal.

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