FDA Grants Fast Track Designation to Factor XIa Inhibitor Milvexian to Treat Stroke, ACS, and AF

The investigational factor XIa (FXIa) inhibitor milvexian (Janssen Pharmaceuticals, Johnson & Johnson, Beerse, Belgium; Bristol Myers Squibb, New York, NY) was granted Fast Track designation by the Food and Drug Administration (FDA) for 3 indications under the phase 3 Librexia program. The indications investigated under this comprehensive clinical development program include stroke, acute coronary syndrome (ACS), and atrial fibrillation (AF). Approximately 50,000 participants will take part in the Librexia program, which seeks to examine the effectiveness of milvexian in reducing thrombotic events with no increased risk of bleeding compared with standard therapies. Previous phase 2 trials suggest milvexian has positive efficacy and bleeding profile in stroke patients in whom factor Xa (FXa) inhibitors are not indicated.

Librexia STROKE (NCT05702034), a phase 3, randomized, double-blind, parallel-group, placebo-controlled study with an estimated 15,000 participants, is investigating milvexian’s safety and efficacy when used in combination with a single or dual antiplatelet therapy as a preventative for stroke after a high-risk transient attack or acute ischemic stroke. Librexia ACS (NCT05754957), a randomized, double-blind, placebo-controlled, event-driven study with an estimated 16,000 participants, is evaluating milvexian’s safety and efficacy when used after recent ACS. Librexia AF (NCT05754957), a randomized, double-blind, double-dummy, parallel group, active-controlled study with an estimated 15,500 participants, is assessing the safety and efficacy of milvexian in treating AF as compared to Eliquis (apixaban, Bristol Myers Squibb, New York, NY), an FXa inhibitor.

"For milvexian to receive Fast Track Designation from the FDA for all three indications demonstrates the enormous unmet need that still exists for the treatment of thrombotic events, like heart attack and stroke," said James F. List, MD, PhD, Global Therapeutic Area Head at Janssen Pharmaceuticals.

Fast Track Designation is associated with expedited development and review of drugs designed to treat serious medical conditions and fill unmet medical needs.