PEDFIC 11,2
Double-blind, randomized, placebo-controlled
45
sites
15
countries
participated
Bylvay
40 mcg/kg/day
(n=23)
Bylvay
120 mcg/kg/day
(n=19)
Placebo
(n=20)
Randomization 1:1:1
(N=62)
24 weeks
Primary endpoint5,6
Proportion of participant's positive pruritus assessments over 24 weeks*
Secondary endpoints included5,6
- Proportion of participants with serum bile acid (sBA) response at week 24†
- Change in sleep parameters through week 24‡
PEDFIC 21,3,4
Open-label extension
41
sites
15
countries
participated
COHORT 1A (continued from PEDFIC 1)
Bylvay 
Bylvay 120 mcg/kg/day
(n=35)
COHORT 1B (continued from PEDFIC 1)
Placebo Bylvay 120 mcg/kg/day
(n=19)
COHORT 2 (newly enrolled)
Bylvay 120 mcg/kg/day
(n=17)
72 weeks
Primary endpoint4,7
Proportion of participant's positive pruritus assessments over 72 weeks*
Secondary endpoints included4,7
Change from baseline in sBA at week 72
Exploratory endpoint4,7
Change in sleep parameters through week 72‡
Enrolled PEDFIC 1 participants were as young as 6 months of age with1,5
- Clinical genetic confirmation of PFIC1 or PFIC2
- Elevated sBA
- History of significant pruritus
Concomitant ursodeoxycholic acid (UDCA) and other antipruritic medications were permitted, provided they were at a stable dose at least 4 weeks before enrollment with no dosage changes planned during study period.4-7
Enrolled PEDFIC 2 participants included those continuing from PEDFIC 1 and newly enrolled Cohort 2 participants4
- Any age
- Any PFIC type
- Elevated sBA
- History of significant pruritus
Concomitant ursodeoxycholic acid (UDCA) and other antipruritic medications were permitted, provided they were at a stable dose at least 4 weeks before enrollment with no dosage changes planned during study period.4-7
- *Defined as a scratching score of ≤1 or at least a 1-point reduction from baseline on the PRUCISION™ ObsRO instrument.4,5
- †sBA response was defined as a 70% decrease in sBA or a final sBA level of ≤70 μmol/L (28.6 μg/mL).5,6
- ‡Data reported using the PRUCISION™ ObsRO instrument; caregivers answered up to 7 questions about the effects that itch had on the participant’s sleep and sleep-related parameters twice daily.4-7
In the PEDFIC 1 and PEDFIC 2 trials,
Pruritus was measured using the PRUCISION™ ObsRO scale8
- A validated 5-point scale from 0 (“No scratching”) to 4 (“Worst possible scratching”)1,8
- Scratching scores were recorded twice daily by a caregiver1,8
- All participants had medium or worse scratching at baseline (ObsRO score ≥2)1,8
The PRUCISION™ ObsRO scale is a validated instrument that defines a ≥1-point reduction as a clinically meaningful change in pruritus score8
Bylvay is approved for the treatment of pruritus in all PFIC types1§
§Limitation of Use: Bylvay may not be effective in a subgroup of PFIC type 2 patients with specific ABCB11 variants resulting in non-functional or complete absence of the bile salt export pump protein.
ObsRO=observer-reported outcomes; PFIC=progressive familial intrahepatic cholestasis.
References:
- Bylvay Prescribing Information. Cambridge, MA: Ipsen Biopharmaceuticals, Inc.; 2024.
- ClinicalTrials.gov. A double-blind, randomized, placebo-controlled, phase 3 study to demonstrate efficacy and safety of A4250 in children with progressive familial intrahepatic cholestasis types 1 and 2 (PEDFIC 1). NCT03566238. Updated September 5, 2021. Accessed April 23, 2023.
- 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.
- Thompson RJ, Artan R, Baumann U, et al. Interim results from an ongoing, open-label, single-arm trial of odevixibat in progressive familial intrahepatic cholestasis. JHEP Rep. 2023;5(8):100782. doi:10.1016/j.jhepr.2023.100782
- 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(9):830-842.
- Data on file A4250-005. Boston, MA: Albireo Pharma, Inc.
- Data on file A4250-008. 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(11):5105-5125.