Rye Brook, NY – June 11, 2018 – The U.S. Food and Drug Administration (FDA) decision to expand the approval of a targeted therapy for patients with chronic lymphocytic leukemia (CLL) and small lymphocytic leukemia (SLL) is a significant development in the treatment of these patients, according to The Leukemia & Lymphoma Society® (LLS).
The FDA approved venetoclax (Venclexta®) to treat patients with CLL, the most common leukemia in adults, and a closely related blood cancer, SLL, with or without a mutation called 17p deletion, who have relapsed or did not respond after at least one prior therapy. The therapy is now approved for use in combination with the antibody therapy, rituximab (Rituxan®), or alone as a monotherapy.
The expanded approval of this targeted therapy gives patients the opportunity to take an oral, chemotherapy-free treatment regimen that might allow patients to stop their treatment after about two years.
The FDA had previously approved venetoclax in 2016 for relapsed and refractory CLL and SLL patients only in cases where they have a rare subset known as 17p deletion, in which a piece of chromosome 17 is missing. This is a very high-risk group of patients with a poor prognosis.
More than 126,000 patients in the U.S. currently live with CLL, a typically slow-moving blood cancer that begins in the bone marrow. Of those CLL patients who do not respond to therapy or who have relapsed, approximately 30 percent are found to have this particular mutation.
Since 2002, LLS has supported more than $15 million in research leading to the development of this treatment through its collaborative Specialized Center of Research (SCOR) grant program. The research team is led by Jerry Adams, Ph.D., at Walter and Eliza Hall Institute of Medical Research in Melbourne, Australia. Andrew Roberts, M.D., M.S., Ph.D., a member of Dr. Adams’ SCOR team, was one of the clinicians who led studies of the pivotal Phase 2 clinical trial of venetoclax upon which the FDA based its original approval.
“We’re proud to say that research we have long supported has yielded this important treatment option for patients with CLL and SLL,” said LLS President and CEO Louis J. DeGennaro, Ph.D. “Our track record of advancing groundbreaking therapies for the blood cancers is paying off in our impact for patients. We’ve seen a number of new therapies approved for CLL in recent years, including another innovative therapy, ibrutinib, and more progress is on the horizon.”
Venetoclax, an oral medication, works by targeting the B-cell lymphoma 2 (BCL-2) protein, which supports cancer cell growth and is overexpressed in many patients with CLL. The approval is based on a study that showed patients receiving venetoclax, in combination with rituximab, had a 92 percent overall response rate (ORR) compared to a 72 percent ORR among those given chemotherapy and rituximab. Patients on this regimen were 81 percent less likely to experience progression of their disease progression than those on the chemotherapy regimen.
LLS is already supporting critical research to advance venetoclax by driving forward understanding of resistance to this therapy to ultimately improve and inform the development of new therapies. LLS is also currently supporting research investigating venetoclax as a therapy for patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL) and mantle cell lymphoma (MCL).
To learn more about this approval, please see the FDA’s announcement here.
About The Leukemia & Lymphoma Society
The Leukemia & Lymphoma Society® (LLS) is the world's largest voluntary health agency dedicated to fighting blood cancer. The LLS mission: Cure leukemia, lymphoma, Hodgkin's disease and myeloma, and improve the quality of life of patients and their families. LLS funds lifesaving blood cancer research around the world, provides free information and support services, and is the voice for all blood cancer patients seeking access to quality, affordable, coordinated care.
Founded in 1949 and headquartered in Rye Brook, NY, LLS has chapters throughout the United States and Canada. To learn more, visit LLS.org. Patients should contact the Information Resource Center at (800) 955-4572, Monday through Friday, 9 a.m. to 9 p.m. ET.