Variant invests in developing orphan drugs that improve health outcomes and quality of life for patients with rare diseases.
* FDA agreement to progress directly to Phase 2a
Our evolving product pipeline is targeted to the $100+ billion orphan drug market. Our lead orphan drug candidate is hydroxypropyl beta cyclodextrin (HPβCD) for chronic treatment of two orphan indications, VAR 200: Focal Segmental Glomerulosclerosis (FSGS) and VAR 300: Alport Syndrome (AS). Both of these conditions are rare progressive forms of kidney disease associated with accumulation of cholesterol and lipids, contributing to impaired kidney function, ultimately leading to dialysis and/or kidney transplantation. HPβCD entraps and removes lipids that can cause injury to the kidneys, and it is expected to delay progression of disease and prevent the need for dialysis and/or transplantation in patients with FSGS and AS. Additional orphan indications for HPβCD are under review for near term development. Our longer-term plans include potential development of HPβCD for prevention of diabetic kidney disease (VAR 400).