sBA reductions with Bylvay over time1-3
Change in sBA: ASSERT 1 Through ASSERT 2 Week 241-4
Change in sBA: ASSERT 1 Through ASSERT 2 Week 241-4
- Reductions in sBA were seen as early as
week 4 and sustained through week 242 - Patients who received Bylvay from weeks 1 to 24 had further reductions in sBA by week 482,3
Secondary endpoint: Change in serum bile acid levels.
ASSERT 2 is an ongoing open-label extension trial. Data shown are interim data.1,5-7
Limitations:
The correlation between sBA and clinical outcomes is not known. sBA levels do not accurately predict bile acids in the liver.
The clinical trial was not designed to make conclusions regarding the clinical benefit of sBA reductions. It is not known whether the sBA assay was qualified to assess sBA levels during the analysis.
Reductions in sBA were seen as early as week 45
Clinical trial criteria defined sBA response with a strict cutoff of ≥70% reduction or concentration of ≤70 μmol/L8
At Week 128
Nearly
4 out of 10
Bylvay patients were sBA responders*
37% on Bylvay 120 mcg/kg/day (n=13)
31% with placebo (n=5)
Through Week 248
More than
4 out of 10
Bylvay patients were sBA responders*
46% on Bylvay 120 mcg/kg/day (n=15)
25% with placebo (n=4)
71% and 52%
of Bylvay patients achieved sBA reductions of ≥30% at weeks 12 and 24, respectively (n=25, n=17)8
Limitations:
Data are from a post hoc analysis from a secondary endpoint that was not adjusted for multiplicity. ASSERT 2 is ongoing. Therefore, results should be interpreted with caution.
The correlation between sBA and clinical outcomes is not known. sBA levels do not accurately predict bile acids in the liver.
The clinical trial was not designed to make conclusions regarding the clinical benefit of sBA reductions. It is not known whether the sBA assay was qualified to assess sBA levels during the analysis.
*95% CI: Week 12: placebo (11.02, 58.66), Bylvay 120 mcg/kg/day (21.47, 55.08). N=51; week 24: placebo (7.27, 52.38); Bylvay 120 mcg/kg/day (28.11, 63.65). N=49.8
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Explore the safety profile of Bylvay
ALGS=Alagille syndrome; sBA=serum bile acid.
References:
- Data on file A4250-012. November 10, 2022. Boston, MA: Albireo Pharma, Inc.
- Data on file A4250-012 Tables-Figures. Boston, MA: Albireo Pharma, Inc.
- Data on file A4250-015 Table 14.2.2.1, sBA Change. Boston, MA: Albireo Pharma, Inc.
- Data on file A4250-015. November 11, 2022. Boston, MA: Albireo Pharma, Inc.
- Bylvay Prescribing Information. Boston, MA: Albireo Pharma, Inc.; 2023.
- 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 Percent sBA Responders, Weeks 12 and 24. Boston, MA: Albireo Pharma, Inc.
- Data on file A4250-012. November 10, 2022. Boston, MA: Albireo Pharma, Inc.
- Data on file A4250-012 Tables-Figures. Boston, MA: Albireo Pharma, Inc.
- Data on file A4250-015 Table 14.2.2.1, sBA Change. Boston, MA: Albireo Pharma, Inc.
- Data on file A4250-015. November 11, 2022. Boston, MA: Albireo Pharma, Inc.
- Bylvay Prescribing Information. Boston, MA: Albireo Pharma, Inc.; 2023.
- 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 Percent sBA Responders, Weeks 12 and
24. Boston, MA: Albireo Pharma, Inc.