FDA Grants First Breakthrough Therapy Designations to Cystic Fibrosis Drugs

The Breakthrough Therapy initiative, which is part of the 2012 Food and Drug Administration Safety and Innovation Act, will bring promising treatments more quickly through an expedited review process.

The Breakthrough Therapy initiative, which is part of the 2012 Food and Drug Administration Safety and Innovation Act, will bring promising treatments more quickly through an expedited review process.

The FDA granted the first-ever Breakthrough Therapy Designations to 2 cystic fibrosis medications from Vertex Pharmaceuticals. The first designation was given to Kalydeco (ivacaftor), a drug that is already approved by the FDA to treat patients 6 years and older who have the G551D mutation. This drug was given the designation for its potential new indications. The other designation was given to Kalydeco in combination with investigational agent VX-809 to treat cystic fibrosis patients who have 2 copies of F508del, the most common mutation in cystic fibrosis.

According to the FDA, a breakthrough drug is one that “is intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition and for which preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.” Janet Woodcock, MD, director of FDA’s Center for Drug Evaluation and Research, noted that the Breakthrough Therapy Designation will complement the FDA’s 3 other efforts to expedite drug development and review: Fast Track, Priority Review, and Accelerated Approval.

Although Vertex said in a release that the “implications of Breakthrough Therapy Designation cannot be determined at this time,” Colorado United States Senator Michael Bennet (D, CO), said that many cystic fibrosis patients who are waiting for a successful treatment option will welcome the news. “Now, FDA will work with Vertex to move these potentially lifesaving treatments through the FDA’s approval process quickly and safely—and hopefully they’ll be in the hands of patients in the near future,” Bennet said on his website. “Our goal for the breakthrough designation is that it will help bring more and more lifesaving cures to patients more efficiently than ever before.”

Currently there is no formal guidance in place for drugs that could be potential candidates for the Breakthough Designation, but the FDA plans to release guidance on this designation and other expedited drug development pathways in the near future. Until that time, drug companies that believe they have a groundbreaking molecule on their hands can submit their application to the FDA following the criteria outlined on their website.

According to Jeff Allen, executive director of Friends of Cancer Research, Kalydeco was among many of the drugs the group studied in a historic review of drugs that may be considered groundbreaking therapies. Allen said that many drug manufacturers will be looking closely at Kalydeco moving forward as a result of its new designation. “I think Kalydeco is an example where it has altered the course of medical practices,” Allen noted, saying that the designation proves that there is a general consensus about the success of Vertex’s new drug.