FDA Grants Fast Track Designation to Star Therapeutics’ VGA039 for von Willebrand Disease

Image Credit: Adobe Stock Images/Катерина Євтехова

The FDA has granted Fast Track Designation to Star Therapeutics’ VGA039, an investigational monoclonal antibody targeting Protein S for the treatment of von Willebrand disease (VWD). According to the company, the treatment is being developed as a universal, subcutaneous (SC) hemostatic therapy for all types of VWD, offering a convenient dosing regimen. Further, the designation marks the first drug candidate to receive this status for VWD, potentially accelerating its path to market.¹

“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, founder, Star Therapeutics, in a press release. “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.”

In June 2023, Star Therapeutics presented promising clinical data from a Phase I study in health volunteers. Presented at the 32^nd Congress of the International Society on Thrombosis and Haemostasis, results found that the pharmacokinetics and pharmacodynamics for VGA039 support convenient SC dosing and showed nearly 100% SC bioavailability, with a favorable safety and tolerability profile. Additionally, there were no serious or thromboembolic adverse events observed, as well as infusion-related or injection site reactions.

“We welcome the ongoing progress of VGA039 as the potential first subcutaneous treatment for VWD to reduce the substantial treatment burden that currently impacts VWD patients and families,” said Jeanette Cesta, executive director, VWD Connect Foundation, in a June 2024 press release. “We join the patient community in encouraging the advancement of new treatments that continue to bring us hope for the future.”

Previous results from an oral presentation at the American Society of Hematology in December 2022 found that VGA039 demonstrated in vivo efficacy in a VWD non-human primate model. Further, it demonstrated in vitro the ability to restore thrombin generation in numerous bleeding disorders, supporting the potential of VGA039 to be effective as a universal hemostatic agent.²

“We are delighted to present these first clinical data for VGA039. VGA039’s high subcutaneous bioavailability and favorable pharmacokinetic profile supports convenient subcutaneous dosing for VWD prophylaxis,” said Gary Patou, MD, chief medical officer, Star Therapeutics, in the June press release. “We are excited to continue moving ahead with our VIVID clinical program of VGA039 in VWD patients.”

According to the Centers for Disease Control and Prevention (CDC), VWD occurs with equal frequency among men and women, affecting up to 1% of the general population. Despite the equal frequency, women are more likely to experience symptoms of VWD because of the increased bleeding it causes during their menstrual periods, during pregnancy, and after childbirth. Between 2012 and 2023, more than 33,456 men, women, and children were seen at hemophilia treatment centers for treatment of VWD.

According to a CDC survey of women with VWD, there is an average of 16 years between the first sign of bleeding symptoms and diagnosis of a bleeding disorder. Additionally, women have reported an average of six bleeding symptoms before a diagnosis has been made. Further, 38% of women reported that they were first diagnosed by a hemophilia treatment center physician, with 42% reporting that they were treated by a hematologist, 28% by an internist or a family physician, and 19% by a gynecologist.³

References

1. Star Therapeutics Receives FDA Fast Track Designation for VGA039 in Von Willebrand Disease (VWD). Business Wire. January 6, 2025. Accessed January 6, 2025. https://www.businesswire.com/news/home/20250106373894/en

2. Star Therapeutics Presents Clinical Data for VGA039, a Subcutaneous Therapy for the Treatment of Von Willebrand Disease (VWD). Star. June 26, 2024. Accessed January 6, 2024. https://star-therapeutics.com/star-therapeutics-presents-clinical-data-for-vga039-a-subcutaneous-therapy-for-the-treatment-of-von-willebrand-disease-vwd/

3. Data and Statistics on von Willebrand Disease. CDC. Accessed January 6, 2024. https://www.cdc.gov/von-willebrand/data/index.html#:~:text=Prevalence-,Von%20Willebrand%20disease%20(VWD)%20occurs%20with%20equal%20frequency%20among%20men,during%20pregnancy%2C%20and%20after%20childbirth.