The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to VT3989, a novel approach for the treatment of mesothelioma that blocks a process critical to cells growing and dividing. According to a news release from the drug’s manufacturer, Vivace Therapeutics, this experimental treatment represents a promising new approach for patients currently facing extremely limited options.
Mesothelioma Clinical Trial Results Show Encouraging Activity Profile
VT3989 treats mesothelioma by blocking a cellular process called palmitoylation in a group of proteins known as TEAD. These proteins are essential components of the Hippo pathway, which regulates how the aggressive form of cancer’s cells grow and divide. When this pathway malfunctions, it can lead to the uncontrolled cell growth and cancer development that make mesothelioma so difficult to treat.
The FDA’s orphan drug approval comes after the manufacturer demonstrated encouraging clinical activity and a favorable safety profile in its ongoing open-label phase 1 study of more than 200 patients with solid tumors, including patients with mesothelioma whose cancer had progressed after receiving chemotherapy and immunotherapy treatments. Those patients specifically received VT3989 in combination with the immunotherapy drugs Opdivo (nivolumab) and Yervoy (ipilimumab),
Mesothelioma Drug Development Benefits from Orphan Designation Status
The FDA’s orphan drug designation was created for the specific purpose of encouraging the development of treatments for rare diseases affecting fewer than 200,000 people in the U.S., which includes mesothelioma. It provides substantial benefits, including tax credits for eligible clinical trials, FDA fee exemptions, and up to seven years of market exclusivity following regulatory approval. These incentives have driven pharmaceutical companies to invest in developing treatments for rare conditions that might otherwise be commercially unattractive.
“The granting of orphan drug designation to VT3989 underscores the critical need for new, effective therapies for mesothelioma, an aggressive cancer with limited treatment options,” said Sofie Qiao, president and chief executive officer of Vivace Therapeutics. The company issued a statement stressing its continued commitment to advancing VT3989 through clinical development and its plans to transition into a registrational phase 3 study for mesothelioma with the FDA by the end of 2025.
Innovative drugs and continued investment in finding a cure offer new hope to patients facing a mesothelioma diagnosis. For information about other resources available to you, contact the Patient Advocates at Mesothelioma.net today at 1-800-692-8608.
