At Retrophin, our mission is to deliver life-changing therapies to people living with rare diseases, and particularly to patients with the greatest medical needs – those who have few, if any, treatment options.

Giving patients a chance, providing hope, is what drives us.

We are a fully integrated biopharmaceutical company focused on advancing the development of our lead pipeline assets: sparsentan for focal segmental glomerulosclerosis (FSGS), a serious kidney disorder that often leads to end-stage renal disease;1 and RE-024 for pantothenate kinase associated neurodegeneration (PKAN), a life-threatening neurological disorder that typically begins in early childhood.2These assets have pioneering potential, as sparsentan could be the first FDA-approved pharmacological treatment for FSGS, and RE-024 could be the first approved replacement therapy targeting the underlying cause of PKAN.

In late 2016, we announced positive results from the Phase 2 DUET study in which the overall sparsentan treatment group achieved statistical significance in the study's primary efficacy endpoint, reduction of proteinuria.3 We are currently working with the U.S. Food and Drug Administration (FDA) to find the most expeditious path forward for sparsentan. In parallel, we are working to initiate the Phase 3 FORT study of RE-024, and expect to begin dosing patients in the coming months. The study will be conducted according to a Special Protocol Assessment (SPA) agreement with the FDA. As part of this process, Retrophin and the agency have agreed that the design of this pivotal trial is adequate to support a New Drug Application seeking U.S. approval of RE-024 for the treatment of PKAN.

We are also advancing a liquid formulation of ursodeoxycholic acid for the treatment of a chronic liver disease called primary biliary cholangitis (PBC). In 2017, we anticipate filing a New Drug Application with the FDA and if approved, plan to make liquid ursodeoxycholic acid available to the subset of PBC patients who have difficulty swallowing.


These activities are complemented by our research exploring the potential of early-stage assets in several rare diseases. We have conducted early-stage research for RE-034, a novel synthetic analog of the ACTH hormone, and are exploring strategic development options for this candidate, including external partnerships and out-licensing opportunities. In addition, we are currently working with leaders in patient advocacy and academia on the development of a novel therapeutic for patients with NGLY1 deficiency.


Our focus on R&D is supported by revenues from our commercial products: Thiola® (tiopronin) tablets, Cholbam® (cholic acid) capsules, and Chenodal® (Chenodiol Tablets 250 mg). Since acquiring rights to Thiola®, we have more than doubled access to this medication for patients with cystinuria and work closely with the community to ensure all who are medically eligible for Thiola® have affordable access to it. Cholbam® is the first and only FDA-approved treatment for people with bile acid synthesis disorders and Zellweger spectrum disorders. Chenodal® is considered by the FDA to be medically necessary for the treatment of cerebrotendinous xanthomatosis (CTX), a rare, progressive, and underdiagnosed lipid storage disease affecting many parts of the body.4,5

Our goal is to ensure that patients have an opportunity to achieve the best possible therapeutic outcomes, and we strive to meet their unique needs by investing in broader access to our medications. Our Total Care HUB® eases access to Thiola®, Cholbam® and Chenodal® by offering personalized and comprehensive support services – at no cost to patients, physicians or insurance companies. As part of this service, dedicated pharmacists and HUB counselors are available to answer product questions, and assist with insurance verification, reimbursement options, and financial support.

We also work closely with the patient community to ensure that everyone who is medically eligible for our therapies has affordable access to them. We offer co-pay assistance and free medication to patients who qualify, as part of our patient assistance program. We also understand the challenges of receiving an accurate diagnosis and do our part by sponsoring free cholestatic genetic testing for qualified patients to help diagnose a variety of disorders.

Headquartered in San Diego with offices in Cambridge, MA and Dublin, Ireland, we are approximately 150 employees strong and structured to meet the unique needs of people living with rare diseases. Integrity, Accountability, Compassion, Teamwork, Scientific Excellence, and Entrepreneurial Spirit are the values that guide our work each day and what we expect from one another.

Giving patients a chance, providing hope, is what drives us.



  1. Middleton, et al. Nephrology Rounds 2007; 5(4).
  2. Gregory, et al. GeneReviews. 2002.
  3. Trachtman H, Nelson P, Radko K on behalf of the DUET Investigators. Efficacy and Safety of Sparsentan, a Dual Angiotensin II and Endothelin Type A Receptor Antagonist, in Patients with Focal Segmental Glomerulosclerosis: A Phase 2 Trial (DUET).  Presented at the American Society of Nephrology Kidney Week Meeting; November 15-20 2016, Chicago, IL.
  4. Data on file. San Diego, CA: Retrophin, Inc.; July 2016.
  5. Lorincz, et al. Arch Neurol 2005;62(9):1459-1463.