We are impatient for the patients we serve.
We are driven to deliver safe and effective therapies to patients as quickly as possible. To do this, we are advancing a pipeline of potential first-in-class medicines targeting rare diseases with significant unmet needs and oftentimes no approved treatment options. Our efforts are led by late-stage development programs of sparsentan, which is a novel product candidate targeting rare kidney diseases – focal segmental glomerulosclerosis (FSGS) and IgA nephropathy (IgAN). We are also partnering with leaders in patient advocacy and the National Institutes of Health (NIH) on earlier-stage development work in rare liver diseases. In addition, pegtibatinase (TVT-058), the first potential disease-modifying therapy for the treatment of classical homocystinuria (HCU), is in Phase 1/2 development.
Today we aim to help patients with our four approved commercial therapies, Chenodal® (chenodiol), Cholbam® (cholic acid), Thiola® (tiopronin), and Thiola EC® (tiopronin) delayed-release tablets. We are committed to broad access for our FDA-approved therapies, and we provide personalized clinical support services through the Total Care Hub. We also sponsor diagnostic testing at no cost to qualified patients to help accurately diagnose a variety of disorders.