Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Volixibat · 2 trials · 3 indications
The Adult ItchRO is an 11-point NRS measurement of itch severity ranging from 0=no itch to 10=worst possible itch.
| Arm | Type | Description |
|---|---|---|
| Part 1 Arm 1: Volixibat 20mg | EXPERIMENTAL | Participants randomized to this arm will receive volixibat 20mg twice daily. |
| Part 1 Arm 2: Volixibat 80mg | EXPERIMENTAL | Participants randomized to this arm will receive volixibat 80mg twice daily. |
| Part 1 Arm 3: Placebo | PLACEBO_COMPARATOR | Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily. |
| Part 2 Arm 1: Volixibat Selected Dose 20mg | EXPERIMENTAL | Participants randomized to this arm will receive volixibat 20mg twice daily. |
| Part 2 Arm 2: Placebo | PLACEBO_COMPARATOR | Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily. |
| Name | Type | Description |
|---|---|---|
| Volixibat | DRUG | Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor. |
| Placebo | DRUG | Capsules matched to study drug without the active pharmaceutical ingredient |
Inclusion Criteria: * Provide signed informed consent at the screening visit as well as comply with all study visits and requirements through the end of the study * Male or female, age ≥18 years at the screening visit * Confirmed diagnosis of PBC in line with the AASLD guidelines * UDCA, anti-pruri...
Volixibat is an investigational small molecule being developed for itching caused by Primary Sclerosing Cholangitis (PSC) and Primary Biliary Cholangitis (PBC). It is currently in Phase 2 clinical trials for both conditions.
Volixibat is a small molecule that targets the apical sodium-dependent bile acid transporter (ASBT). By inhibiting this transporter, it aims to reduce bile acid absorption and alleviate itching associated with cholestatic liver diseases.
Volixibat is being developed by Mirum Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker MIRM.
Volixibat is in Phase 2 clinical development. It is not yet approved by the FDA and remains investigational, with active trials ongoing for both Primary Sclerosing Cholangitis and Primary Biliary Cholangitis.
Volixibat is being studied in two Phase 2 trials: NCT04663308 for itching in Primary Sclerosing Cholangitis with 182 participants, and NCT05050136 for itching in Primary Biliary Cholangitis with 333 participants. Both are randomized, double-blind, placebo-controlled studies.
Volixibat is a distinct investigational drug and is not known to be the same as any other marketed medication. It is being studied specifically for pruritus associated with cholestatic liver diseases.