Regeneron Pharmaceuticals on Wednesday said the FDA has accepted its evinacumab filing for priority review as an adjunct to other lipid-lowering therapies in patients with homozygous familial hypercholesterolaemia (HoFH). According to the company, evinacumab is the first ANGPTL3 blocker to show efficacy in patients with HoFH, including those with little to no LDL receptor function. A target action date for the drug, which was previously designated a breakthrough therapy by the FDA for HoFH, has been set for February 11, 2021.
Regeneron estimates that HoFH affects approximately 1300 patients in the US, and while guidelines recommend early and intensive lowering of LDL cholesterol, patients with the condition are less responsive, or even unresponsive, to standard lipid-lowering therapies, including statins and PCSK9 inhibitors. The company currently markets the PCSK9 inhibitor Praluent (alirocumab) in the US for adults with primary hyperlipidaemia, including heterozygous familial hypercholesterolaemia.
Significant LDL lowering
The FDA filing for evinacumab is backed by data from the Phase III ELIPSE HoFH trial, which were presented at the American College of Cardiology's Annual Scientific Session together with the World Congress of Cardiology (ACC.20) earlier this year. The study achieved its primary endpoint, with evinacumab-treated patients reducing their baseline levels of LDL cholesterol by 49% compared to placebo at 24 weeks. Further, 47% of patients in the evinacumab arm saw their LDL cholesterol cut down to under 100 mg/dL, despite entering the trial with average levels of 260 mg/dL on other lipid-lowering therapies.
Regeneron also pointed to research published in the NEJM in 2017 showing that patients with loss-of-function mutations in their ANGPTL3 gene have significantly lower levels of key blood lipids, including LDL cholesterol. "By blocking the ANGPTL3 protein, evinacumab was designed to replicate this loss-of-function mutation effect to lower LDL cholesterol in patients with HoFH," the company said.
EU filing progressing
Meanwhile, the drugmaker noted that submissions for evinacumab are also progressing in the EU, where in June the European Medicines Agency's Committee for Medicinal Products for Human Use recommended an accelerated assessment for the therapy.
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