Focusing on making a difference in rare diseases with significant unmet need
| Program | Therapeutic Area | Preclinical | Phase 1 | Phase 2 | Phase 3 |
|---|---|---|---|---|---|
|
Sparsentan1 |
IgAN | ||||
|
Sparsentan |
FSGS | ||||
|
CDCA2 |
CTX | ||||
|
Pegtibatinase (TVT-058)3 |
HCU | ||||
|
ALGS Collaboration |
ALGS |
1 On February 17, 2023, Travere announced that the FDA granted approval of sparsentan under the accelerated pathway for the reduction of proteinuria in IgAN. Read the press release>
2 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.
3 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