OUR Pipeline
Focusing on making a difference in rare diseases with significant unmet need
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. **TVT-058 is currently in a Phase 1/2 clinical study.
FSGS: focal segmental glomerulosclerosis; IgAN: IgA nephropathy; CTX: cerebrotendinous xanthomatosis; HCU: classical homocystinuria; NGLY1 deficiency: N-glycanase 1 deficiency; ALGS: Alagille syndrome
We are involved in numerous clinical trials for our pipeline product candidates targeting rare chronic conditions. Find out which ones are enrolling and visit our clinical trial websites.