At Retrophin, our mission is to identify, develop and deliver life-changing therapies to people living with rare diseases.
This is what we do every day in our jobs by working hard, supporting our team members and living our values: Accountability, Entrepreneurial Spirit, Integrity, Patient Focus, Scientific Excellence and Teamwork. We strive to help our organization succeed by pursuing our vision – to become a preeminent, global and fully-integrated biopharmaceutical company within the rare disease community that is dedicated to giving patients a chance and providing hope.
Our research and development (R&D) centers on our promising pipeline featuring product candidates targeting rare diseases:
- Fosmetpantotenate is currently being evaluated in a pivotal Phase 3 trial (FORT) (PKANFORTStudy.com) which, if successful, has the potential to be the first approved replacement therapy for pantothenate kinase-associated neurodegeneration (PKAN), a life threatening neurological disorder that typically begins in early childhood
- Sparsentan is a novel product candidate with dual mechanism of action, currently being evaluated in a Phase 3 study (DUPLEX) (FSGSDUPLEX.com) which, if successful, has the potential to be the first FDA-approved pharmacologic treatment for focal segmental glomerulosclerosis (FSGS) and IgA nephropathy (IgAN), disorders characterized by progressive scarring of the kidney often leading to end-stage renal disease.
- CNSA-001, for the treatment of phenylketonuria (PKU), a rare, genetic metabolic condition that can lead to neurological and behavioral impairment; CNSA-001 is advancing in development through a strategic research collaboration.
We are also moving forward with a liquid formulation of ursodeoxycholic acid for primary biliary cholangitis (PBC), a chronic liver disease, as well as working with leaders in patient advocacy and public research to identify potential therapeutics for NGLY1 deficiency, a progressive neurological disorder with no approved therapeutic options.
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 Chenodal, Cholbam and Thiola by offering personalized and comprehensive support services – at no cost to patients, physicians or insurance companies. Dedicated pharmacists and HUB counselors are available to answer product questions; assist with insurance verification, reimbursement options and financial support; and provide co-pay assistance and free medication to patients who qualify. We also sponsor free cholestatic genetic testing for qualified individuals to help accurately diagnose a variety of disorders.
With experienced, knowledgeable and compassionate team members, Retrophin is a thriving and patient-centric company striving to become a leader in the rare disease community.