FDA Approves Lerodalcibep, Third-Generation PCSK9 Inhibitor, to Treat Hypercholesterolemia, Including HeFH

The FDA announced its approval of lerodalcibep-liga (Lerochol; LIB Therapeutics) as an adjunct to diet and exercise to reduce low-density lipoprotein cholesterol (LDL-C) in adults with hypercholesterolemia, including those with heterozygous familial hypercholesterolemia (HeFH). LIB Therapeutics expects the injectable therapy to reach the US market in spring 2026, according to a company statement.

Lerodalcibep is a novel, third-generation PCSK9 inhibitor designed for once-monthly, self-administered subcutaneous use. “The primary challenge in lipid management today is helping patients achieve and maintain the increasingly stringent LDL-C targets in current guidelines,” Dean J. Kereiakes, MD, chairman of The Christ Hospital Heart and Vascular Institute and professor of medicine at the University of Cincinnati, said in a statement.

“While PCSK9 inhibitors as a class deliver powerful cholesterol lowering potential, [lerodalcibep] was designed to address the barriers that have limited their use." Kereiakes included ease of use features like a single small monthly injection, self-administered at home with extended room-temperature stability, as some of those obstacles. The temperature stability is particulary important for home storage and for individuals who travel. "For patients with cardiovascular disease who require lifelong cholesterol management,[lerodalcibep] offers an important addition to our treatment options.”

Phase 3 Clinical Trial Program

The FDA based its decision on data from the global phase 3 LIBerate Clinical Trial Program, which included more than 2,900 participants with established cardiovascular disease (CVD), individuals at high or very high CVD risk, and those with heterozygous and homozygous familial hypercholesterolemia (HeFH, HoFH, respectively).

Across the registration-enabling, placebo-controlled trials, participants self-administered lerodalcibep once monthly for up to 52 weeks. More than 2,400 participants continued treatment in a 72-week open-label extension study.

LIB Therapeutics reported that lerodalcibep produced sustained LDL-C reductions of 60% or greater in the CVD and CVD risk groups. In participants with HeFH and more severe baseline LDL-C elevations, reductions reached 50% or more, according to the company. Safety and tolerability were reported as generally favorable, with no treatment-related serious adverse events observed in the long-term extension studies.

Unmet Need in Lipid Management

Despite widespread statin use and the availability of additional lipid-lowering therapies, many adults fail to reach guideline-recommended LDL-C targets, the company observed. Evidence continues to link lower LDL-C levels with improved CV outcomes.

Familial hypercholesterolemia contributes substantially to this unmet need, affecting approximately 1 in 200 individuals worldwide. The genetic condition is characterized by markedly elevated LDL-C levels from birth and requires lifelong management. Although PCSK9 inhibitors offer potent LDL-C lowering, access challenges and treatment burden have limited their adoption in routine practice, according to LIB Therapeutics. “[Lerodalcibep] was designed for patients who need life-long treatment to achieve and maintain the new lower LDL-C goals especially those with cardiovascular disease and the millions with inherited high cholesterol, (familial hypercholesterolemia or FH),” Evan Stein, MD, PhD, founder, CEO, and chief scientific officer of LIB said in a statement.

Availability and Access

LIB Therapeutics plans to launch LEROCHOL in the United States as a prefilled syringe in spring 2026. The company expects to introduce an upgraded autoinjector device later in the year. LIB said it will work with payers, clinicians, and patient advocacy organizations to support access and will provide details on patient assistance programs at launch.

Stein acknowledged the scope of the development effort behind the approval. “I wish to thank the more than 2,500 patients throughout the world who participated in our clinical trials and our small team of very dedicated colleagues at LIB who believed in the drug and worked tirelessly for nearly 10 years, including during the COVID-19 pandemic, to achieve this milestone,” he said.

Source: US Food and Drug Administration approves LIP Therapeutics Lerochol (lerodalcibep-liga) for adults with elevated LDL cholesterol. News release. LIP Therapeutics. December 15, 2025. Accessed December 18, 2025. https://www.businesswire.com/news/home/20251215907781/en/U.S.-Food-and-Drug-Administration-Approves-LIB-Therapeutics-LEROCHOL-lerodalcibep-liga-for-Adults-with-Elevated-LDL-Cholesterol