FDA approves Regeneron's Evkeeza for ultra-rare form of high cholesterol

Regeneron Pharmaceuticals said the FDA approved Evkeeza (evinacumab-dgnb) as an adjunct to other LDL cholesterol-lowering therapies to treat adult and paediatric patients aged 12 years and older with homozygous familial hypercholesterolaemia (HoFH). The drug becomes the first treatment cleared in the US that blocks the function of the ANGPTL3 protein regulating plasma lipid levels.

The filing, assessed under a priority review, was backed by data from the Phase III ELIPSE HoFH trial. Results showed that patients treated with the once-monthly intravenous infusion saw their LDL cholesterol levels fall an average 49% from baseline after six months, compared to placebo participants who received other lipid-lowering therapies alone, meeting the primary endpoint.

The study also found that patients who took Evkeeza achieved an average 135 mg/dL reduction in LDL cholesterol, compared to a decrease of 3 mg/dL for placebo. Regeneron noted that similar lowering was observed in difficult-to-treat patients who often do not respond to other therapies because of limited LDL receptor function. Moreover, Evkeeza-treated subjects saw significant reductions on key secondary measures, such as ApoB, non-HDL cholesterol and total cholesterol, compared to placebo.

Average cost of $450,000 per patient

Daniel Rader, who was involved with Evkeeza clinical trials, suggested the ANGPTL3 blocker could be "a potentially transformational new treatment for people with HoFH," as existing medications for this population "are insufficient for the majority of patients." Regeneron estimates HoFH affects approximately 1300 patients in the US. The company noted that as Evkeeza is administered based on weight, the average wholesale acquisition cost per is expected to be approximately $450,000 per year.

Regeneron currently markets the PCSK9 inhibitor Praluent (alirocumab) in the US for adults with primary hyperlipidaemia, including heterozygous familial hypercholesterolaemia. The company and its partner Sanofi, which sells Praluent outside the US, won a favourable US appeals court ruling this week in an ongoing patent battle with Amgen over PCSK9 inhibitors.

Meanwhile, Regeneron said Evkeeza is under review by regulators in the EU, with the European Medicines Agency's Committee for Medicinal Products for Human Use last year recommending an accelerated assessment based on high unmet medical need and therapeutic innovation demonstrated by the drug.

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