Retrophin, Inc., a biopharmaceutical company, focuses on the development, acquisition, and commercialization of therapies for the treatment of serious, catastrophic, or rare diseases. Its product line includes Cholbam, a cholic acid for the treatment of bile acid synthesis disorders due to single enzyme defects, and for adjunctive treatment of peroxisomal disorders, such as Zellweger spectrum disorders in patients who exhibit manifestations of liver disease, steatorrhea, or complications from decreased fat soluble vitamin absorption; Thiola, which is indicated for the prevention of cystine stone formation in patients with severe homozygous cystinuria; and Chenodal, a synthetic oral form of chenodeoxycholic acid for radiolucent stones in well-opacifying gallbladders. The company's product candidates include Sparsentan for the treatment of focal segmental glomerulosclerosis, which is a cause of end-stage renal disease and nephrotic syndrome; RE-024, a novel small molecule for the treatment of pantothenate kinase-associated neurodegeneration; and RE-034, a synthetic hormone analog. Retrophin, Inc. has research collaboration with the Grace Wilsey Foundation and the Warren Family Research Center for drug discovery and development at the University of Notre Dame. Retrophin, Inc. was founded in 2011 and is headquartered in San Diego, California.
News: https://www.thestreet.com/story/136...li-advances-drug-for-rare-kidney-disease.html Retrophin (RTRX) announced positive results from a mid-stage clinical trial Wednesday in which patients with a rare kidney disease responded to treatment with its experimental drug sparsentan at more than twice the rate of patients receiving a control drug. The company's next step will be to meet with the U.S. Food and Drug Administration to map out plans for a pivotal phase III study of sparsentan, with the goal of securing the drug's approved for the treatment of focal segmental glomerulosclerosis, or FSGS.
