Focusing on making a difference in rare diseases with significant unmet need
Program | Therapeutic Area | Preclinical | Phase 1 | Phase 2 | Phase 3 | Approved |
---|---|---|---|---|---|---|
FILSPARI® (sparsentan)1 |
IgAN | Preclinical |
Phase 1 |
Phase 2 |
Phase 3 |
|
Sparsentan2 |
FSGS | Preclinical |
Phase 1 |
Phase 2 |
Phase 3 |
|
Pegtibatinase (TVT-058)3 |
HCU | Preclinical |
Phase 1 |
Phase 2 |
Phase 3 |
|
ALGS Collaboration |
ALGS | Preclinical |
Phase 1 |
Phase 2 |
Phase 3 |
|
1 On February 17, 2023, we announced that the FDA granted approval of sparsentan under the accelerated pathway for the reduction of proteinuria in IgAN. Read the press release>
2 On May 1, 2023, we announced topline primary efficacy results from the pivotal Phase 3 DUPLEX Study of sparsentan. Read the press release>
3 Pegtibatinase (TVT-058) is currently in a Phase 1/2 clinical study.
FSGS: focal segmental glomerulosclerosis; IgAN: IgA nephropathy; HCU: classical homocystinuria; ALGS: Alagille syndrome
*On September 5, 2023, Travere Therapeutics announced that Mirum Pharmaceuticals acquired all of Travere’s rights and assets related to Cholbam® and Chenodal®. For more information, please visit mirumpharma.com.