FDA Fast Track Designation for VMX-C001 Highlights Need for Improved Anticoagulant Reversal

Tatiana Shepeleva - stock.adobe.com

FDA has granted fast track designation to VMX-C001, an investigational therapy developed by VarmX, a biotech company based in The Netherlands, to bypass the effects of Factor Xa direct oral anticoagulants (FXa DOACs), the company announced on Sept. 3, 2025. The designation underscores the growing demand for new treatment strategies to manage life-threatening bleeding and urgent surgery in patients taking anticoagulants. VarmX specializes in the development of innovative approaches for the bypass of FXa DOACs and inherited coagulation disorders (1).

Fast track designation is typically reserved for therapies targeting serious or life-threatening conditions that have the potential to address unmet medical needs. For developers of these therapies, the designation provides enhanced communication with FDA, the option of rolling review for a biologics license application, and the possibility of priority review, which may shorten the path to patient access.

“Receiving FDA fast track designation is a strong recognition of the real unmet need for treatments that can rapidly restore coagulation to enable urgent surgery in patients on Factor Xa direct oral anticoagulants, as well as the potential of our novel bypass agent approach,” said John Glasspool, CEO, VarmX, in a company press release (1).

What’s the clinical development plan and mechanism of action for this therapy?

The fast track designation follows FDA’s clearance of an investigational new drug application for VMX-C001 in July 2025, enabling the launch of a pivotal Phase III global trial (EquilibriX-S) (2). The study is designed to assess whether the therapy can rapidly and durably restore coagulation in patients who require emergency surgery while on FXa DOACs.

VMX-C001 is a modified human factor X protein engineered to be insensitive to FXa DOACs. By bypassing their anticoagulant activity, the compound aims to reestablish the coagulation cascade. Its design incorporates several clinical advantages: It is suitable for universal dosing regardless of which FXa DOAC a patient has received, allows for rapid administration, is compatible with commonly used anticoagulants such as heparin, and avoids increasing thrombotic risk (1).

These features offer significant advantages to developers of these types of therapies. For instance, universal dosing and compatibility with standard anticoagulation regimens reduce complexity in emergency settings, where speed and predictability are critical. The absence of added thrombotic risk could also address a longstanding safety concern with reversal strategies.

How does VMX-C001 address a growing clinical burden in this therapeutic space?

The burden of anticoagulant-related emergencies is expected to rise sharply. By 2030, around 30 million patients in the United States, Europe, and Japan are projected to be prescribed FXa DOACs for conditions such as atrial fibrillation and prevention of deep vein thrombosis. Each week, more than 25,000 of these patients either suffer severe bleeding episodes or face urgent surgical procedures where uncontrolled bleeding presents a serious risk (3,4).

“This FDA fast track designation significantly strengthens the position of VMX-C001 to potentially become a new option for the substantial number of patients on FXa DOACs who need emergency surgery,” Glasspool said in the release.

For the bio/pharmaceutical industry, the fast track designation of VMX-C001 illustrates how innovation in protein engineering and clinical trial design can address urgent therapeutic gaps. The designation also reflects regulatory recognition of the challenges posed by widespread anticoagulant use, an area likely to remain a priority as patient populations age and chronic cardiovascular conditions rise.

References

1. VarmX. VarmX receives FDA Fast Track Designation for lead asset VMX-C001. Press Release. Sept. 3, 2025.
2. VarmX. VarmX receives IND approval for Phase 3 trial of VMX-C001 in Urgent Surgery. Press Release. July 8, 2025.
3. Polaris Market Research. Direct Oral Anticoagulants (DOACs) Device Market Size, Share, Trends, & Industry Analysis Report by Product Type, by Disease Indication, by Application, by Patient Demographic, by End User, and by Region—Market Forecast, 2025–2034. Market Research Report. June 2025. https://www.polarismarketresearch.com/industry-analysis/direct-oral-anticoagulants-doacs-device-market
4. Lee, S. R.; Lee, K. Y.; Park, J. S.; et al. Perioperative Factor Xa Inhibitor Discontinuation for Patients Undergoing Procedures with Minimal or Low Bleeding Risk. JAMA Netw Open. 2025, 8 (2), e2458742. DOI: 10.1001/jamanetworkopen.2024.58742