OUR PIPELINE

Focusing on making a difference in rare diseases with significant unmet need

Program Therapeutic Area Preclinical Phase 1 Phase 2 Phase 3

Sparsentan

IgAN

Sparsentan

FSGS

CDCA*

CTX

Pegtibatinase (TVT-058)**

HCU

ALGS Collaboration

ALGS

THERAPEUTIC AREAS

*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