FDA Grants Star Two Key Designations for VWD Therapy

Rare Daily Staff

The U.S. Food and Drug Administration granted Star Therapeutics Rare Pediatric Disease and Breakthrough Therapy designations for its experimental therapy VGA039 for von Willebrand disease, a common inherited bleeding disorder.

Von Willebrand disease, or VWD, affects how blood clots and can lead to frequent or prolonged bleeding. The condition is caused by low levels or dysfunction of von Willebrand factor, a protein that helps platelets stick together and form clots. Patients may experience symptoms ranging from frequent nosebleeds and easy bruising to severe bleeding during surgery or injury.

VGA039 is a monoclonal antibody designed to address those challenges. The therapy targets Protein S and works through two mechanisms: promoting platelet attachment and enhancing fibrin formation, both of which help restore normal blood clotting. Unlike existing options, VGA039 is designed for once-monthly, self-administered injections under the skin, potentially reducing treatment burden and improving convenience. Current preventive treatments often require multiple intravenous infusions each week, which can be burdensome for patients and caregivers.

The therapy is currently being evaluated in a late-stage phase 3 clinical trial involving adolescent and adult patients.

The FDA’s designations are intended to speed development and create incentives for promising therapies. Rare Pediatric Disease designation is granted to treatments targeting serious conditions affecting fewer than 200,000 people in the United States, primarily in children. If the drug is approved, the company may receive a Priority Review Voucher, which can be used to accelerate FDA review of another product or sold to another company.

Breakthrough Therapy designation is reserved for drugs that show substantial improvement over existing treatments based on early clinical evidence. It allows for closer collaboration with the FDA and can shorten development and review timelines.

The designations for VGA039 build on earlier Fast Track status and are supported by mid-stage clinical data showing meaningful reductions in bleeding episodes across different types of VWD, according to the company.

Star Therapeutics said it is continuing to enroll patients aged 12 and older in its ongoing global Phase 3 trial as it works toward potential regulatory approval.