Rare Disease Report

FDA Approves Development of VAR 200 for Treatment of FSGS

APRIL 16, 2018
Krista Rossi
Today, Variant Pharmaceuticals, Inc announced that the US Food and Drug Administration (FDA) has approved the development of VAR 200 (2-hydroxypropyl-β-cyclodextrin, or HPβCD) for the treatment of focal segmental glomerulosclerosis (FSGS) in adults.
 
VAR 200 (2-hydroxypropyl-β-cyclodextrin, or HPβCD) is a phase 2a-ready asset designed to minimize or prevent kidney cell damage and maintain adequate kidney function in patients with FSGS, 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.
 
Stephen C. Glover, Variant's Co-founder, Chairman, and Chief Executive Officer, stated his excitement for the drug’s development approval. "We are pleased that the FDA concurred with our plans to progress directly into a phase 2a clinical trials in adult patients with FSGS, which will shorten our development time significantly. With no currently available disease-specific treatments approved for FSGS, a high percentage of patients progress to end-stage renal disease within 10 years, requiring dialysis and/or kidney transplant, which has a high recurrence rate."1 

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
 
"Based on our productive Pre-IND meeting with the FDA, we can confirm that the IND filing will not require any additional nonclinical data before proceeding directly to the phase 2a trial in FSGS adults,” added Glover. “Likewise, we are working with FDA to establish parameters for clinical development of VAR 200 in pediatrics, and we have obtained a clearer understanding of our path forward for longer-term phase 2b/3 trials in adults.”

VAR 200 is also believed to possess the potential to induce remission of proteinuria and delay FSGS progression; this would fulfil an unmet need in this population.

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References:
  1. Variant Pharmaceuticals Completes Pre-IND Meeting with FDA on VAR 200 for Focal Segmental Glomerulosclerosis (FSGS), a Rare Kidney Disease. Prnewswire.com. https://www.prnewswire.com/news-releases/variant-pharmaceuticals-completes-pre-ind-meeting-with-fda-on-var-200-for-focal-segmental-glomerulosclerosis-fsgs-a-rare-kidney-disease-300629892.html. Accessed April 16, 2018.
  2. About Hydroxypropyl Beta Cyclodextrin.variantpharma.com. https://www.variantpharma.com/pipeline/about-hydroxypropyl-beta-cyclodextrin. Accessed April16, 2018. 


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