FDA Accepts BLA for Lerodalcibep, Novel PCSK9 Inhibitor to Lower LDL-C

The US Food and Drug Administration (FDA) has accepted for review a Biologics License Application (BLA) for lerodalcibep to reduce low-density lipoprotein cholesterol (LDL-C) across a broad patient population, according to a news release from LIB Therapeutics.¹

The FDA has set a Prescription Drug User Fee Act action date of December 12, 2025, and does not plan to hold an advisory committee meeting to discuss the application, the company said.¹

Lerodalcibep is a novel small protein–binding, third-generation PCSK9 inhibitor for adults with atherosclerotic cardiovascular disease (ASCVD) or at very high or high risk for ASCVD, including those with heterozygous familial hypercholesterolemia (HeFH), and for individuals aged 10 years or older with homozygous familial hypercholesterolemia (HoFH). Lerodalcibep has been developed as a once-monthly, single small-volume, subcutaneous injection that remains stable at room temperature, offering an alternative to currently approved PCSK9 inhibitors that require refrigeration.¹

"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," said Evan Stein, MD, PhD, FACC, the co-founder, Chief Operating and Scientific Officer of LIB Therapeutics, in a news release. "Lerodalcibep has demonstrated robust and sustained long-term LDL cholesterol-lowering, enabling the vast majority of patients to achieve these more stringent lower LDL-cholesterol targets with excellent safety and adherence across clinical trials. We look forward to working with regulators to make Lerodalcibep available to patients around the world.”¹

The BLA submission is supported by data from a clinical trial program of approximately 2900 patients, including five phase 3 registration trials (the LIBerate program). More than 2300 patients who required additional LDL-C lowering beyond maximally tolerated statins and other oral agents were included; an additional 2400 patients have continued in a 72-week, open-label extension study.¹

LIB Therapeutics anticipates filing a Marketing Authorization Application for lerodalcibep with the European Medicines Agency in the second quarter of 2025. If approved, lerodalcibep would enter a market of expanding PCSK9 inhibitor therapies, which have become integral in efforts to achieve lower LDL-C targets for patients at high cardiovascular risk.

“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,” said David Cory, Chief Executive Officer of LIB Therapeutics, in the news release. “The LIB operating team is now focused on Lerodalcibep U.S. commercial launch preparation. Lerodalcibep will enter a now rapidly growing global PCSK9 market on target to reach $5B in 2025 and projected to be $10B by 2030.”¹

Reference

1. LIB Therapeutics announces FDA acceptance of Biologics License Application for lerodalcibep to lower LDL-cholesterol across broad patient population [news release]. Business Wire. Published February 10, 2025. Accessed February 10, 2025. https://www.businesswire.com/news/home/20250210806330/en/LIB-Therapeutics-Announces-FDA-Acceptance-of-Biologics-License-Application-for-Lerodalcibep-to-Lower-LDL-Cholesterol-Across-Broad-Patient-Population