Reductions in sBA were seen as early as week 42
Clinical trial criteria defined sBA response with a strict cutoff of ≥70% reduction or concentration of ≤70 μmol/L3
At Week 243,4
4 out of 10
Bylvay patients were sBA
responders*
44% on Bylvay 40 mcg/kg/day (n=23)
21%on Bylvay 120 mcg/kg/day (n=19)
vs 0% with placebo (n=20)
Through Week 481
4 out of 10
Bylvay patients treated for 48 weeks were sBA responders†
42% on Bylvay 120 mcg/kg/day (n=66)
Interim data from an ongoing study.
sBA reductions as low as 30% are above the natural variation in PFIC5
62%
and
77%
of Bylvay patients achieved sBA reductions of ≥30% at weeks 48 and 96, respectively (n=66, n=22)1‡
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 reduction. It is unknown whether the sBA assay was qualified to assess sBA levels during the analysis.
*95% CI: placebo (0.00, 16.84); Bylvay 40 mcg/kg/day (23.19, 65.51); Bylvay 120 mcg/kg/day (6.05, 45.57). N=62.
†Average of values from weeks 46-48. All patients were transitioned to Bylvay 120 mcg/kg/day at week 24. N=111.
‡Average of values from weeks 46-48, 94-96. Pooled data from the completed PEDFIC 1 and ongoing PEDFIC 2
phase 3 studies in patients with PFIC through a data cutoff of January 31, 2022.
sBA response in patients with mild vs moderate hepatic impairment6
45%
n/N=27/60
and
24%
n/N=8/34
While sBA response was observed across all levels of hepatic impairment, 45% of patients with mild impairment at baseline had a response vs 24% with more severe impairment
sBA reductions with Bylvay over time7
Change in sBA: PEDFIC 1
Change in sBA: PEDFIC 1 Through PEDFIC 2 Week 727
Change in sBA: PEDFIC 2 Week 727
Reductions in serum bile acids were seen as early as week 4 and sustained through week 967
Secondary endpoint: Reduction in serum bile acid.
PEDFIC 2 is an ongoing open-label extension trial. Data shown are interim data.2,3,8,9
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 reduction. It is unknown whether the sBA assay was qualified to assess sBA levels during the analysis.
See the safety and tolerability data for Bylvay
References:
- Data on file A4250-ISE_T122_sBA Resp. November 15, 2022. Boston, MA: Albireo Pharma, Inc.
- Bylvay Prescribing Information. Boston, MA: Albireo Pharma, Inc.; 2023.
- Thompson RJ, Arnell H, Artan R, et al. Odevixibat treatment in progressive familial intrahepatic cholestasis: a randomised, placebo-controlled, phase 3 trial. Lancet Gastroenterol
Hepatol. 2022;7:830-842. - Data on file A4250-005. Boston, MA: Albireo Pharma, Inc.
- Data on file MED-21-00176 sBA Natural Variation. October 2021. Boston, MA: Albireo Pharma, Inc.
- D’Antiga L, Özen H, Lainka E, et al. Hepatic impairment
classifications at baseline in responders to odevixibat therapy in children with progressive
familial intrahepatic
cholestasis. Presented at: 2022 NASPGHAN/CPNP/APGNN Annual Meeting; October 12-15, 2022; Orlando, FL, USA. - Data on file PEDFIC 1 and 2 Figure Pru and sBA. 2022. Boston, MA: Albireo Pharma, Inc.
- Data on file A4250-008. Boston, MA: Albireo Pharma, Inc.
- ClinicalTrials.gov. An open-label extension study to evaluate long-term efficacy and safety of A4250 in children with progressive familial intrahepatic cholestasis types 1 and 2
(PEDFIC 2). NCT03659916. Updated October 12, 2022. Accessed April 23, 2023.