PFIC: Safety

In the Bylvay treatment group^1,2

PEDFIC 1: Serious Adverse Events (SAEs)¹

Placebo N=20	Bylvay 40 mcg/kg/day N=23	Bylvay 120 mcg/kg/day N=19
5	0	3

In the Bylvay treatment group^1,2

All SAEs were unrelated to treatment, and none led to discontinuation
- Treatment-emergent SAEs in the
  placebo group included: insomnia,
  weight stagnation, itching, influenza,
  influenza H1N1, urinary tract infection,
  viral upper respiratory tract infection,
  viral gastroenteritis, auricular
  hematoma, viral illness, pyrexia, prurigo
  nodularis
- Treatment-emergent SAEs in the
  odevixibat 120 mcg/kg/day group
  included: supraventricular tachycardia,
  urinary tract infection, dehydration,
  elevated liver function tests
2 patients discontinued, 1 due to treatment- emergent adverse events (in the Bylvay 120 mcg/kg/day group)
No deaths were reported

Clinical adverse events in PEDFIC 1^1,3

PEDFIC 1: Clinical Adverse Reactions (≥2%)³

Preferred Term	Placebo (N=20) n (%)	Bylvay 40 mcg/kg/day (N=23) n (%)	Bylvay 120 mcg/kg/day (N=19) n (%)
Diarrhea	2 (10)	9 (39)	4 (21)
Transaminases increased (ALT, AST)	1 (5)	3 (13)	4 (21)
Vomiting	0	4 (17)	3 (16)
Abdominal pain	0	3 (13)	3 (16)
Blood bilirubin increased	2 (10)	3 (13)	2 (11)
Fat-soluble vitamin deficiency (A, D, E)	1 (5)	0	3 (16)
Splenomegaly	0	0	2 (11)
Cholelithiasis	0	0	1 (5)
Dehydration	0	0	1 (5)
Fracture	0	1 (4)	0

Most treatment-emergent adverse events were mild to moderate in severity
Most common adverse reactions (>2%) were liver test abnormalities, diarrhea, abdominal pain, vomiting, and fat-soluble vitamin deficiency
In PEDFIC 2, treatment-emergent adverse reactions were similar as observed in PEDFIC 1. The most common reason for Bylvay treatment interruption was liver test abnormalities (increases in ALT, AST, direct and total bilirubin)

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ALT=alanine aminotransferase; AST=aspartate aminotransferase; PFIC=progressive familial intrahepatic cholestasis; SAE=serious adverse event.

References:

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.
Bylvay Prescribing Information. Boston, MA: Albireo Pharma, Inc.; 2023.