The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the investigational von Willebrand disease (VWD) therapy VGA039. The therapy is being developed by Star Therapeutics, a biotechnology company based in San Francisco, CA.
VGA039 is a subcutaneously-delivered monoclonal antibody therapy designed to target Protein S as a means of restoring proper blood clotting in people with all types of VWD.
Subcutaneous (Sub-Q) therapies require an injection just under the skin, as opposed to intravenously administered products which necessitate venous access. As an easier to administer therapy, Sub-Q treatments have the advantage of greater convenience which in turn may increase rates of adherence.
The new fast track designation follows recent reporting of positive interim clinical trial data for VGA039, which was presented during the 66th American Society of Hematology meeting in December 2024. FDA’s Fast Track designation is meant to facilitate the development and expedite the review of drugs that treat a broad range of serious conditions and address an unmet medical need. The purpose is to get newer drugs in clinical development through FDA’s approval process faster and ultimately to the patient earlier.
If VGA039 should receive FDA approval, it would be the first Sub-Q therapy to be available to treat all types of VWD.
“VGA039 is the first drug candidate to receive Fast Track designation for VWD, and we are committed to advancing drug innovation for this debilitating disease that has lagged behind other bleeding disorders,” said Adam Rosenthal, PhD, CEO and Founder of Star Therapeutics. “Fast Track designation enables us to potentially accelerate the development path for VGA039 as a therapy that can transform the way VWD is treated with a convenient, subcutaneous therapy for patients with all types of VWD.”
Learn more about Fast Track criteria and processes on the FDA website.
Source: Businesswire, January 8, 2025
