FDA Approved Development of VAR 200 for Treatment of FSGS

Variant Pharmaceuticals, Inc received US Food and Drug Administration (FDA) has approval for the development of VAR 200 (2-hydroxypropyl-β-cyclodextrin, or HPβCD) for the treatment of focal segmental glomerulosclerosis (FSGS) in adults. [1]

FSGS is a rare kidney disease that causes cholesterol and lipids to accumulate in the part of the kidneys that filters waste from the blood. VAR 200’s intended action is believed to be achieved through the trapping and removal of excess intracellular cholesterol and lipids. Additionally, VAR 200 also has the potential to treat other kidney conditions associated with the damaging effects of intracellular accumulation of cholesterol and lipids, such as Alport syndrome.

In preclinical studies with mice, HPβCD showed promising results. In mouse models of focal segmental glomerulosclerosis FSGS and Alport Syndrome, the progression of kidney disease was prevented, and diabetic kidney disease was prevented or partially prevented in two separate pre-clinical studies.²

[1] https://www.raredr.com/news/fda-approves-development-var-200-treatment-fsgs