Focusing on making a difference in rare diseases with significant unmet need
*CDCA is not indicated for CTX, but has received a medical necessity determination in the US by the FDA for CTX. Travere Therapeutics is conducting a Phase 3 clinical trial to examine the safety and efficacy of CDCA (Chenodal®) for the treatment of CTX.
**Pegtibatinase (TVT-058) is currently in a Phase 1/2 clinical study.
FSGS: focal segmental glomerulosclerosis; IgAN: IgA nephropathy; CTX: cerebrotendinous xanthomatosis; HCU: classical homocystinuria; ALGS: Alagille syndrome