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.
In pursuing our patient-centered purpose, the Retrophin Rare Disease Network at Children’s National Health System in Washington, D.C. was created through a multi-year financial commitment to support the work of the Children’s National Rare Disease Institute (CNRDI). The innovative, global “hub and spoke” model utilized by CNRDI aims to improve patient access, standardize care models, and establish worldwide best practices in the diagnosis and treatment of rare diseases.
CNRDI is a first-of-its-kind center focused exclusively on advancing the care and treatment of children and adults with rare genetic diseases. It is the first National Organization for Rare Disorders (NORD)-designated Center of Excellence, and provides a medical home for patients and families seeking the most advanced care and expertise for rare genetic conditions that remain largely unknown to the general medical community.
In our ongoing efforts to support caregivers of people living with rare disease, Retrophin worked with NORD to establish the Rare Caregiver Respite Program, a first-of-its-kind program aimed at providing caregivers of rare disease patients a much-needed respite.
Our pipeline features late-stage development programs targeting rare diseases with significant unmet medical needs including sparsentan 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. In partnership with leaders in patient advocacy and government research, we are also working to identify potential therapeutics for NGLY1 deficiency and Alagille syndrome, conditions with no approved treatment options. Our R&D efforts are supported by revenues from our four commercial products Chenodal® (chenodiol), Cholbam® (cholic acid), Thiola® (tiopronin) and THIOLA ECTM(tiopronin) delayed-release tablets.
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.
Through our portfolio of approved products and promising pipeline focused on hepatology and nephrology, we strive to help people achieve the best possible outcomes today and tomorrow. But most importantly, it is the commitment of our experienced, knowledgeable and compassionate team members, who put patients at the center of everything they do, that truly matters.