ASSERT is the only pivotal study of an IBAT inhibitor in ALGS to enroll patients with1-3
- JAG1 mutations (48 participants, or 92.3%)
- NOTCH2 mutations (4 participants, or 7.7%)
ASSERT participants were as young as 12 months to 16 years with1-4
- Genetically confirmed diagnosis of ALGS
- History of significant pruritus
- Elevated sBA levels
See the impact on pruritus, sleep, and sBA levels
ALGS=Alagille syndrome; IBAT=ileal bile acid transporter; sBA=serum bile acid.
References:
- Bylvay Prescribing Information. Boston, MA: Albireo Pharma, Inc.; 2023.
- Kamath BM, Stein P, Houwen RHJ, Verkade HJ. Potential of ileal bile acid transporter inhibition as a therapeutic target in Alagille syndrome and progressive familial intrahepatic cholestasis. Liver Int. 2020;40(8):1812-1822.
- Data on file A4250-012. November 10, 2022. Boston, MA: Albireo Pharma, Inc.
- ClinicalTrials.gov. A phase 3 double-blind, randomized, placebo-controlled study of the safety and efficacy of odevixibat (A4250) in patients with Alagille syndrome (ASSERT). NCT04674761. Updated April 10, 2023. Accessed April 24, 2023.
- Bylvay Prescribing Information. Boston, MA: Albireo Pharma, Inc.; 2023.
- Kamath BM, Stein P, Houwen RHJ, Verkade HJ. Potential of ileal bile acid transporter inhibition as a therapeutic target in Alagille syndrome and progressive familial intrahepatic cholestasis. Liver Int. 2020;40(8):1812-1822.
- Data on file A4250-012. November 10, 2022. Boston, MA: Albireo Pharma, Inc.
- ClinicalTrials.gov. A phase 3 double-blind, randomized,
placebo-controlled study of the safety and efficacy of
odevixibat (A4250) in patients with Alagille syndrome
(ASSERT). NCT04674761. Updated April 10, 2023.
Accessed April 24, 2023.