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 approval pathway for the reduction of proteinuria in IgAN. On September 21, 2023, we announced confirmatory data from the Phase 3 PROTECT Study of FILSPARI®. 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, we announced that Mirum Pharmaceuticals acquired all of Travere’s rights and assets related to Cholbam® and Chenodal®. For more information, please visit mirumpharma.com.