Investor Relations

Retrophin, Inc. (NASDAQ:RTRX) is a fully-integrated biopharmaceutical company dedicated to identifying, developing and delivering life-changing therapies to people living with rare diseases. 

The Company’s strategy centers on advancing its pipeline, in particular its programs for two potential first-in-class therapies: fosmetpantotenate for pantothenate kinase associated neurodegeneration (PKAN), a life-threatening neurological disorder that typically begins in early childhood; and sparsentan for focal segmental glomerulosclerosis (FSGS) and IgA nephropathy (IgAN), rare disorders characterized by progressive scarring of the kidney often leading to end-stage renal disease. Research in additional rare diseases is also underway, including a joint development arrangement evaluating the potential of CNSA-001 in phenylketonuria (PKU), a rare genetic metabolic condition that can lead to neurological and behavioral impairment. Retrophin's R&D efforts are supported by growing revenues from the Company's portfolio of three commercial products.