Basel, 07 May 2015
US FDA grants breakthrough therapy designation for investigational Bcl-2 inhibitor venetoclax in 17p deletion relapsed-refractory chronic lymphocytic leukemia
Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the US Food and Drug Administration (FDA) has granted breakthrough therapy designation for venetoclax (RG7601, GDC-0199/ABT-199), an investigational medicine being developed in partnership with AbbVie, for the treatment of people who have relapsed or refractory chronic lymphocytic leukemia (CLL) with a genetic abnormality known as 17p deletion. Breakthrough therapy designation is designed to accelerate the development and review of medicines intended to treat serious and life-threatening diseases with evidence showing that the medicines may provide a substantial improvement over current treatment options.
“People who have relapsed or refractory CLL with a 17p deletion typically have a poor prognosis, and do not respond to many currently available treatment options,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Clinical Development. “We are pleased that the FDA has granted venetoclax Breakthrough Therapy designation and hope this regulatory pathway will help us bring venetoclax to people with this difficult-to-treat disease soon."
About CLL and 17p deletion
CLL is a slow-growing cancer of the blood and bone marrow that is generally considered incurable, and is one of the most common adult leukemias worldwide.1,2 Most cases of CLL (95 percent) start in white blood cells called B-cells.1 In certain cases of CLL, a part of chromosome 17 is lost and along with it an important gene that controls apoptosis called p53.3 The 17p deletion is found in 3 to 10 percent of previously untreated cases and approximately 30 to 50 percent of relapsed or refractory cases.4 People with 17p deletion CLL have poor results with conventional chemotherapy regimens and a median life expectancy of less than three years.5
Venetoclax (RG7601, GDC-0199/ABT-199) is an investigational small molecule designed to selectively bind and inhibit Bcl-2 proteins, an important regulator of a process called programmed cell death, or apoptosis. These proteins are thought to impact how certain blood cancers form, develop and become resistant to treatment. Bcl-2 proteins are expressed at high levels in CLL, non-Hodgkin’s lymphoma (NHL) and in other cancers caused by blood cells called B-cells. Venetoclax is being developed in collaboration with AbbVie and currently being studied in Phase II and Phase III studies for CLL and in Phase I and II studies for several other blood cancers.
About Roche in hematology
For more than 20 years, Roche has been developing medicines with a goal of redefining treatment in hematology. Today, we’re investing more than ever in our effort to bring innovative treatment options to people with diseases of the blood. In addition to approved medicines MabThera/Rituxan (rituximab) and Gazyvaro/Gazyva (obinutuzumab), Roche’s pipeline of investigational hematology medicines includes an anti-CD79b antibody drug conjugate (RG7596/polatuzumab vedotin), a small molecule antagonist of MDM2 (RG7112) and, in collaboration with AbbVie, a small molecule Bcl-2 inhibitor (venetoclax, RG7601/GDC-0199/ABT-199). Roche’s dedication to developing novel molecules in hematology expands beyond oncology, with the development of the investigational hemophilia A treatment ACE910.
Headquartered in Basel, Switzerland, Roche is a leader in research-focused healthcare with combined strengths in pharmaceuticals and diagnostics. Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and neuroscience. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management. Roche’s personalised healthcare strategy aims at providing medicines and diagnostics that enable tangible improvements in the health, quality of life and survival of patients. Founded in 1896, Roche has been making important contributions to global health for more than a century. Twenty-four medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and chemotherapy.
In 2014, the Roche Group employed 88,500 people worldwide, invested 8.9 billion Swiss francs in R&D and posted sales of 47.5 billion Swiss francs. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit roche.com.
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1) Leukemia & Lymphoma Society, “ Chronic Lymphocytic Leukemia.” http://www.lls.org/sites/default/files/file_assets/cll.pdf
2) Leukemia & Lymphoma Society, “Chronic Lymphocytic Leukemia.” http://www.lls.org/leukemia/chronic-lymphocytic-leukemia
3) Selner, L. et al. (2013) “What Do We Do with Chronic Lymphocytic Leukemia with 17p Deletion?” Curr Hemetol Malig Rep. 8(1):81-90.
4) Schnaiter, A. et al. (2013) “17p Deletion in Chronic Lymphocytic Leukemia: Risk Stratification and Therapeutic Approach.” Hematol Oncol Clin N Am 27 (2013) 289–301
5) Stilgenbauer, S, and Zenz, T, (2010) “Understanding and Managing Ultra High-Risk Chronic Lymphocytic Leukemia.” ASH Education Book. 2010(1):481-488.