• Variant Pharmaceuticals Submits Application for U.S. Orphan Drug Designation for VAR 200 (2-Hydroxypropyl-β-Cyclodextrin) To Treat Focal Segmental Glomerulosclerosis (FSGS)

    • November 1, 2018
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    Variant Pharmaceuticals, Inc. (Variant), a clinical stage orphan drug company developing first-in-class drugs for patients with rare diseases, announced today that it has filed an application with the FDA for orphan drug designation for VAR 200 (2-hydroxypropyl-β-cyclodextrin, or 2HPβCD) for treatment of FSGS.  FSGS is a rare kidney disease affecting up to 80,000 people in the U.S. and up to 300,000 people worldwide.

    The Orphan Drug Act was enacted to establish federal government support for development of products for the diagnosis, prevention or treatment of rare diseases or conditions that affect fewer than 200,000 people in the United States. Achieving Orphan Drug Designation facilitates clinical development with assistance throughout the development process from FDA’s Office of Orphan Products Development. This may include tax credits for certain clinical research costs, exemption or waiver of filing fees, and seven-year marketing exclusivity following drug approval.

    “Filing for orphan drug designation is an important step forward in our development plans for VAR 200,” stated Stephen C. Glover, Variant’s Co-founder, Chairman and Chief Executive Officer. “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. With the potential to delay FSGS progression demonstrated in our pre-clinical program, we are anxious to advance our development program for VAR 200 as efficiently and as quickly as possible. Achieving Orphan Drug Designation will support this goal.” 


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