FDA BRIEF: Week of April 11, 2016
VENCLEXTA™ (venetoclax) tablets
Vysis CLL FISH probe kit
AbbVie Inc., North Chicago, Illinois, USA
Genentech, South San Francisco, California, USA
Abbott Molecular, Des Plaines, Illinois, USA
INDICATION: Treatment of patients with chronic lymphocytic leukemia (CLL) with 17p deletion, as detected by an FDA approved test, who have received at least one prior therapy ( approval based on overall response rate; continued approval contingent upon verification in confirmatory trial)
UNMET NEED
- 15,000 new CLL cases diagnosed each year
- 17p deletion lchromosomal abnormality ~ 10%
- Need for new targeted therapy
REG PATHWAY: Breakthrough Therapy Designation, Priority Review, Accelerated Approival, Orphan Drug
MECHANISM OF ACTION: Inhibitor of BCL-2, an antiapoptotic protein
EFFICACY
- Open-label, single-arm, multicenter trial (n=106); patients with CLL with 17p deletion who had received at least one prior therapy
- 17p deletion was confirmed using Vysis CLL FISH Probe Kit
- Primary Endpoint: Overall response rate (ORR) as assessed by an Independent Review Committee (IRC) using the International Workshop for Chronic Lymphocytic Leukemia guidelines : 80.2%
- Median time to first response: 0.8 months
- Median duration of response: 2.9 to 19.0+ months
SAFETY
- Serious complications: Pneumonia, neutropenia with fever, fever, autoimmune hemolytic anemia, anemia and metabolic abnormalities known as tumor lysis syndrome
- Most common side effects: Low white blood cell count (neutropenia), diarrhea, nausea, anemia, upper respiratory tract infection, low platelet count (thrombocytopenia) and fatigue