LIB Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for lerodalcibep, a novel PCSK9 inhibitor aimed at reducing low-density lipoprotein cholesterol (LDL-C) in a broad patient population. The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of December 12, 2025, for its decision. The agency does not plan to hold an advisory committee meeting to discuss the application.
Lerodalcibep is designed for adults with atherosclerotic cardiovascular disease (ASCVD) or those at very high or high risk for ASCVD, including patients with heterozygous familial hypercholesterolemia (HeFH), as well as for individuals aged 10 years and older with homozygous familial hypercholesterolemia (HoFH). The drug is formulated as a convenient once-monthly, self-administered, small-volume subcutaneous injection that does not require refrigeration, offering a more patient-friendly alternative to currently available PCSK9 inhibitors.
Addressing Unmet Needs in Cholesterol Management
According to Dr. Evan Stein, Co-Founder, Chief Operating and Scientific Officer of LIB Therapeutics, "There remains a large unmet need among millions of patients with cardiovascular disease, or at high cardiovascular risk, including the 30 million people with inherited high cholesterol, who are unable to achieve currently recommended guideline-directed goals for LDL cholesterol with current oral therapies." He added that lerodalcibep has demonstrated robust and sustained long-term LDL cholesterol-lowering, enabling the vast majority of patients to achieve more stringent LDL-C targets with excellent safety and adherence in clinical trials.
Clinical Evidence and Trial Data
The BLA submission is supported by data from a comprehensive clinical trial program involving approximately 2,900 patients, including five Phase 3 registration trials known as the LIBerate program. These trials included over 2,300 patients on maximally tolerated statins and other oral agents who required additional LDL-C reduction. Furthermore, more than 2,400 patients have continued into a 72-week open-label extension study.
Data from the Phase 3 LIBERATE-HeFH trial demonstrated a mean placebo-adjusted LDL-C reduction of 65% at 24 weeks with lerodalcibep. Approximately 68% of patients achieved LDL-C reductions of ≥50%, meeting the European Society of Cardiology (ESC) guideline-recommended targets. In the LIBerate-High Risk (HR) trial, lerodalcibep achieved a 56% greater reduction in LDL-C levels than placebo over one year in patients with or at very high risk for CVD.
Convenient Administration and Market Potential
David Cory, Chief Executive Officer of LIB Therapeutics, stated, "Lerodalcibep is a potential best-in-class PCSK9 inhibitor with a once-monthly, single small-volume subcutaneous dose, combined with long ambient stability not requiring refrigeration at home or in travel, offering a more patient-friendly treatment option to achieve the new lower LDL-C goals." Cory also noted that the company is focused on U.S. commercial launch preparation, with lerodalcibep poised to enter a rapidly growing global PCSK9 market expected to reach $5 billion in 2025 and $10 billion by 2030.
Following the FDA BLA filing, LIB Therapeutics anticipates submitting a Marketing Authorization Application to the European Medicines Agency (EMA) in the second quarter of 2025.
