Evkeeza® (Evinacumab) Phase 3 Trial Demonstrates 48% LDL-C Reduction in Children with Ultra-Rare Homozygous Familial Hypercholesterolemia (HoFH)

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Recently, Regeneron announced positive results from a Phase 3 clinical trial evaluating Evkeeza (evinacumab) for the treatment of children aged 5-11 with homozygous familial hypercholesterolemia (HoFH). The trial enrolled 14 children who, despite being on other lipid-lowering therapies, had an average low-density lipoprotein cholesterol (LDL-C) level of 264 mg/dL at the start of the trial—more than twice the target value (<130 mg/dL) for pediatric HoFH patients.

The results showed that the trial met its primary endpoint: adding Evkeeza to other lipid-lowering therapies reduced LDL-C by an average of 48% at week 24. In 79% of patients, LDL-C was reduced by at least half. On average, LDL-C was absolutely reduced by 132 mg/dL from baseline.

All lipid endpoint parameters evaluated in the trial showed decreased levels, typically observed within the first 8 weeks of treatment, including apolipoprotein B, non-high-density lipoprotein cholesterol, lipoprotein A, and total cholesterol. In the trial, Evkeeza was generally well tolerated.

HoFH is an extremely rare genetic disorder and the most severe form of familial hypercholesterolemia (FH). Evkeeza is the first ANGPTL3-targeted therapy approved by the U.S. and EU for adjunctive treatment of HoFH patients aged 12 years and older. Currently, Evkeeza is being evaluated for use in HoFH patients aged 5-11. It is estimated that about 4 in every 1 million people suffer from HoFH, and due to the rarity of patients, the cost of treatment with Evkeeza is substantial, averaging approximately $450,000 per year.

Regeneron's portfolio also includes Praluent, a lipid-lowering therapy for HoFH, which was approved in April last year. Praluent and Evkeeza are currently competing with Amgen’s PCSK9 inhibitor Repatha, which received approval for the HoFH indication before Regeneron. However, Regeneron believes that as the first-in-class therapy with a completely different mechanism of action from PCSK9 inhibitors, Evkeeza should have a competitive edge in the market.

In January this year, Regeneron transferred the rights of Evkeeza in markets outside the United States to Ultragenyx, a rare disease pharmaceutical company. The reasons were, firstly, the desire to focus on the domestic market in the U.S., and secondly, selling a drug for extremely rare diseases abroad posed significant challenges. Analysts at investment bank Jefferies previously noted that the market opportunity for Evkeeza outside the U.S. would not be substantial, with consensus forecasts predicting peak sales in the U.S. market to reach $276 million by 2030.

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Evkeeza® (evinacumab) Phase 3 Trial Demonstrates 48% LDL-C Reduction in Children with Ultra-rare Form of High Cholesterol