FDA Grants Breakthrough Therapy Designation to Novel Therapy for von Hippel-Lindau Disease-Associated RCC

The FDA based its decision on data from a phase 2 trial which showed that MK-6482 induced clinical responses among patients with von Hippel-Lindau disease-associated kidney cancer.

The Food and Drug Administration (FDA) has granted a Breakthrough Therapy Designation for MK-6482, a HIF-2a inhibitor, for the treatment of patients with von Hippel-Lindau (VHL) disease-associated renal cell carcinoma. This breakthrough designation means the therapy will have an expedited regulatory review and development.

“These designations for MK-6482 support the potential of targeting HIF-2a in certain patients with von Hippel-Lindau disease, who currently have limited treatment options and face an increased risk for benign tumors as well as several types of cancer, including renal cell carcinoma,” said Dr. Scott Ebbinghaus, vice president of clinical research at Merck Research Laboratories, in a company-issued press release.

The agency also granted MK-6482 an orphan drug designation, which is part of a mission by the FDA to advance the development of products (or therapies, in this case) that show promise in the diagnosis and/or treatment of a rare disease. von Hippel-Lindau (VHL) disease fits that criteria, as it is a rare inherited mutation that affects approximately 10,000 individuals in the United States.

The two designations were based on data from a phase 2 trial presented at the 2020 ASCO Virtual Scientific Meeting, which demonstrated that treatment with MK-6482 induced an objective response rate of 27.9% in patients with VHL-associated RCC. Additionally, 86.9% of patients had a decrease in the size of their target lesions and the median time to response was 5.5 months.