FDA Grants Fast Track Designation to VERVE-102 Gene Therapy

The US Food and Drug Administration (FDA) has awarded Fast Track designation to VERVE-102 for the treatment of individuals with hyperlipidemia and high cardiovascular risk to lower low-density lipoprotein cholesterol (LDL-C) levels.1

Announced by Verve Therapeutics on April 11, 2025, VERVE-102 is undergoing safety and tolerability assessment in the Phase 1b Heart-2 clinical trial for adults with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease (CAD).

Fast Track designation is granted to advance development and accelerate the review of drugs addressing an unmet medical need for life-threatening conditions.2 Verve Therapeutics can communicate more frequently with the regulatory agency, receive rolling review of an application for marketing approval, and may be eligible for Priority Review during this process.

“Despite available treatment options to lower LDL-C, there remains a pressing need to provide sustained LDL-C lowering and thereby, improve efficacy,” said Sekar Kathiresan, MD, co-founder and chief executive officer of Verve Therapeutics.1 “Nearly 50% of patients discontinue prescribed LDL-C-lowering therapy within a year. Verve has long understood that profound, sustained LDL-C lowering is key to transforming the treatment of atherosclerotic cardiovascular disease (ASCVD).”

The novel in vivo base editing therapy is designed as a single-course dose intended to inhibit the PCSK9 gene to durably lower blood low-density lipoprotein cholesterol (LDL-C). Verve Therapeutics is targeting development initially for HeFH, intending to ultimately treat patients with established ASCVD who continue to be impacted by high LDL-C levels.

The FDA cleared the Investigational New Drug (IND) application for VERVE-102 in March 2025, when the company provided interim data from the dose–escalation part of Heart-2.3

At the cutoff in January 2025, Heart-2 trial data submitted to the FDA included participants across 3 dose cohorts: 0.3 mg/kg, 0.45 mg/kg, and 0.6 mg/kg. The drug was well-tolerated, without observed treatment-related serious adverse events and no clinically significant laboratory abnormalities.

Verve indicated their expectation to report demographic and initial safety and efficacy data from Heart-2 in the second quarter of 2025, with ≥28 days of follow-up for each participant.

The company also announced it remains on track to report final data from the dose-escalation portion of Heart-2, submit the opt-in data package for the PCSK9 program to Eli Lilly and Company, and initiate the Phase 2 clinical trial for the PCSK9 program.1

Verve’s other lead genetic medicine programs each target cholesterol drivers of atherosclerosis, including emnant cholesterol and Lipoprotein(a) [Lp(a)]. VERVE-201 is targeted to permanently shut off the ANGPTL3 gene in patients with refractory hypercholesterolemia with high LDL-C and homozygous familial hypercholesterolemia (HoFH).VERVE-301 is designed to permanently turn off the LPA gene to reduce Lp(a), an independent risk factor for ASCVD.

“As a potential one-dose treatment, we believe VERVE-102 is uniquely designed to address this unmet need,” Kathiresan added.1 “We look forward to collaborating with the FDA as we work to deliver this paradigm-shifting treatment to patients.”

References

1. Verve Therapeutics receives U.S. FDA Fast Track designation for verve-102, an in vivo base editing medicine targeting PCSK9. Verve Therapeutics. April 11, 2025. Accessed April 11, 2025. https://ir.vervetx.com/news-releases/news-release-details/verve-therapeutics-receives-us-fda-fast-track-designation-verve.

2. Commissioner O of the. Fast track. U.S. Food and Drug Administration. Accessed April 11, 2025. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track.

3. Iapoce C. FDA clears ind application for verve-102 gene therapy for HeFH. HCP Live. March 25, 2025. Accessed April 11, 2025. https://www.hcplive.com/view/fda-clears-ind-application-for-verve-102-gene-therapy-for-hefh.