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 pipeline features late-stage development programs targeting rare diseases with significant unmet medical needs.
Fosmetpantotenate is being evaluated in a Phase 3 clinical trial (FORT Study) as a novel replacement therapy for pantothenate kinase-associated neurodegeneration (PKAN), a devastating neurological disorder. In many cases, the first symptoms of PKAN can be seen in early childhood.
Sparsentan is being evaluated in Phase 3 clinical trials as a novel dual acting angiotensin receptor blocker and endothelin receptor antagonist treatment for focal segmental glomerulosclerosis (FSGS), (DUPLEX Study), and IgA nephropathy (IgAN), (PROTECT Study) — disorders characterized by progressive scarring of the kidney often leading to end-stage renal disease.
CNSA-001, an orally bioavailable form of sepiapterin, is being evaluated through a collaboration with Censa Pharmaceuticals for the treatment of phenylketonuria (PKU), a rare, genetic metabolic condition that can lead to neurological and behavioral impairment.
In partnership with leaders in patient advocacy and public research, we are working to identify potential therapeutics for NGLY1 deficiency and Alagille syndrome, rare conditions with no approved treatment options.
We have three FDA-approved products: Chenodal® (chenodiol), Cholbam® (cholic acid) and Thiola® (tiopronin).
We invest in access programs and personalized clinical support services for these therapies through our Total Care Hub at no cost to patients, physicians or insurance companies. Dedicated nurses, pharmacists and HUB counselors are available to answer product questions; assist with insurance verification, reimbursement options and financial support. We also sponsor diagnostic testing at no cost to qualified patients to help accurately diagnose a variety of disorders.