Regulatory Affairs

Regeneron’s evinacumab gets FDA breakthrough therapy status to treat HoFH

PBR Staff Writer Published 07 April 2017

Regeneron Pharmaceuticals has secured breakthrough therapy status from the US Food and Drug Administration (FDA) for its evinacumab to treat patients with homozygous familial hypercholesterolemia (HoFH).

HoFH is an inherited disorder, which can result in premature cardiovascular disease due to high levels of LDL cholesterol.

Evinacumab is an investigational monoclonal antibody to angiopoietin-like protein 3 (ANGPTL3).

ANGPTL3 claimed to serve an inhibitor of lipoprotein lipase and endothelial lipase, in addition to playing a key role in lipoprotein metabolism.

The company is presently planning a phase 3 trial after positive interim phase 2 results for evinacumab in May 2016.

Regeneron’s proof-of-concept study had demonstrated that evinacumab added to current lipid-lowering therapy reduced low-density lipoprotein cholesterol (LDL-C) levels by an additional 55% at week four compared against baseline, the company said.

The single-arm, open label and proof-of-concept study had recruited up to 8 patients with HoFH, who were diagnosed genetically and phenotypically

Regeneron fixed the primary endpoint of the trial as the mean percent change in LDL-C levels from baseline to week four.

The mean reduction in LDL-C in the 4 patients was 55%, after four weeks of treatment and two weeks after administration of the 15 mg/kg IV dose. The percent reductions in LDL-C ranged from 25% to 90% among four patients.

Though rare HoFH disease can occur one to two people per million and those suffering from it can have a risk of premature cardiovascular disease.

Untreated patients can have LDL cholesterol levels ranging from 500 to 1000 mg/dL, compared to normal LDL cholesterol levels of less than 130 mg/dL.

Breakthrough Therapy designation is given to a drug to speed up the development and review of drugs that can treat life-threatening diseases.


Image: The FDA campus at New Hampshire Ave in Silver Spring, Maryland. Photo: courtesy of The U.S. Food and Drug Administration.