Bylvay has been studied in the largest phase 3 clinical trial program in cholestatic pruritus of ALGS and PFIC to date1-3
32
sites
13
countries
participated
A double-blind, randomized, placebo-controlled study with a 24-week treatment period followed by an ongoing,
open-label, 72-week extension
ASSERT 1 • 24 WEEKS1,3
Double-blind, randomized, placebo-controlled
Primary Endpoint 1,3,6
Change in pruritus from baseline to month 6 (weeks 21-24)
Secondary Endpoints 1,3,6
- Change in sBA levels through week 24
- Change in sleep parameters through
week 24 - Change in global symptom relief* at
weeks 4, 12, and 24
ASSERT 2 • 72 WEEKS1,4,5
Ongoing open-label extension
Primary Endpoint4
Change in pruritus through week 72
Secondary Endpoints4
- Change in sBA levels through week 72
- Change in sleep parameters through week 72
- Change in global symptom relief* at weeks 4, 12, 24, 48, and 72
Concomitant UDCA and other antipruritic medications were permitted.6
*Change from baseline in global symptom relief as measured by the patient, caregiver, and clinician on the GIS (PGIS, CaGIS, CGIS) and the GIC (PGIC, CaGIC, CGIC) items.7
Over 50 patients were included in the ASSERT trial1
ALGS=Alagille syndrome; CaGIC=Caregiver Global Impression of Change; CaGIS=Caregiver Global Impression of Symptoms; CGIC=Clinical Global Impression of Change; CGIS=Clinical Global Impression of Symptoms; GIC=Global Impression of Change; GIS=Global Impression of Symptoms; IBAT=ileal bile acid transporter; PFIC=progressive familial intrahepatic cholestasis; PGIC=Patient Global Impression of Change; PGIS=Patient Global Impression of Symptoms; sBA=serum bile acid; UDCA=ursodeoxycholic 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. - 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. - ClinicalTrials.gov. An open-label study to evaluate the long-term safety and efficacy of odevixibat (A4250) in patients with Alagille syndrome (ASSERT-EXT). NCT05035030. Updated
October 18, 2022. Accessed April 24, 2023. - Data on file A4250-015. November 11, 2022. Boston, MA: Albireo Pharma, Inc.
- Data on file A4250-012. November 10, 2022. Boston, MA: Albireo Pharma, Inc.
- Gwaltney C, Ivanescu C, Karlsson L, Warholic N, Kjems L, Horn P. Validation of the PRUCISION instruments in pediatric patients with progressive familial intrahepatic cholestasis. Adv
Ther. 2022;39:5105-5125.
- 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. - 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.
- ClinicalTrials.gov. An open-label study to evaluate the long-term safety and efficacy of odevixibat (A4250) in patients with Alagille syndrome (ASSERT-EXT). NCT05035030. Updated October 18, 2022. Accessed April 24, 2023.
- Data on file A4250-015. November 11, 2022. Boston, MA: Albireo Pharma, Inc.
- Data on file A4250-012. November 10, 2022. Boston, MA: Albireo Pharma, Inc.
- Gwaltney C, Ivanescu C, Karlsson L, Warholic N, Kjems
L, Horn P. Validation of the PRUCISION instruments in
pediatric patients with progressive familial intrahepatic
cholestasis. Adv Ther. 2022;39:5105-5125.