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The First Drug with a Breakthrough Therapy Designation Receives FDA Approval

David M. Fox

David M. Fox,

Washington, D.C.

05 November 2013
On November 1, 2013, the Food and Drug administration (FDA) approved Gazyva (obinutuzumab) for use in combination with chlorambucil to treat patients with previously untreated chronic lymphocytic leukemia (CLL).  CLL is a blood and bone marrow cancer that usually has a slow progression compared to other types of leukemia.  According to the National Cancer Institute, 15,680 Americans will be diagnosed and 4,580 will die from the disease this year.

Gazyva is the first drug with a “breakthrough therapy” designation to receive FDA approval.

Section 506(a) of the Food Drug and Cosmetic Act (FDCA), as added by section 902 of the Food and Drug Administration and Safety and Innovation Act (FDASIA), provides for designation of a drug as a breakthrough therapy if the drug is intended to treat a serious or life-threatening disease and preliminary clinical evidence suggests it provides a substantial improvement over existing therapies.

The designation is intended to expedite the development and review of drugs for serious or life-threatening conditions.  It went into effect on November 1, 2012, and the first breakthrough designations were granted by January 2013.  As of October 25, 2013, FDA lists 92 total requests for breakthrough designation, 29 requests granted, and 41 requests denied.  Gazyva received the designation in May 2013.

The FDA approval of Gazyva, which was granted on the one-year anniversary of the implementation of the breakthrough designation program, was based on a study of 356 individuals in a randomized multicenter clinical trial that compared Gazyva in combination with chlorambucil to chlorambucil alone in participants who had not yet started any treatment for their CLL.  In the study, patients with previously untreated CLL who received Gazyva in combination with chlorambucil had nearly twice as long of a progression-free survival (the amount of time after successful treatment before the CLL returned), an average of 23 months, as those treated with chlorambucil alone, an average of 11.1 months.

"This approval reflects the promise of the Breakthrough Therapy Designation program, allowing us to work collaboratively with companies to expedite the development, review, and availability of important new drugs," said Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA Center for Drug Evaluation and Research, in a press statement.

The author thanks Michael Moskowitz for assistance

David M. Fox

David M. Fox,

Washington, D.C.

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