Table 2.

Changes in serum bile acids and percentage of positive pruritus assessments by PFIC type from PEDFIC 2 baseline through Week 24 of PEDFIC 2.

	Serum bile acids, μmol/L						Proportion of positive pruritus assessments, %
	Baseline		Change from baseline to Week 12		Change from baseline to Weeks 22–24		Initial interval†		Weeks 0–24
	n	Mean (SE)	n	Mean (SE)	n	Mean (SE)	n	Mean (SE)	n	Mean (SE)
PFIC1
Cohort 1A	10	154 (35)	7	-14 (10)	5	-27 (14)	10	27 (10)	7	24 (10)
Cohort 1B	5	206 (28)	4	-40 (53)	3	-82 (31)	5	33 (17)	3	15 (7)
Cohort 2	3	121 (59)	1	-1.5	NA	NA	3	62 (19)	NA	NA
PFIC2
Cohort 1A	24	116 (27)	19	4 (24)	16	-15 (15)	23	24 (6)	19	36 (8)
Cohort 1B	14	294 (37)	11	-157 (48)	8	-167 (65)	13	53 (9)	8	72 (10)
Cohort 2	7	279 (64)	6	-54 (85)	4	-96 (49)	6	62 (14)	3	41 (28)
PFIC3‡
Cohort 2	5	212 (48)	4	-127 (20)	1	-136	5	95 (2)	1	94
MYO5B deficiency‡
Cohort 2	1	169	1	-45	NA	NA	1	87	1	91

All patients in PEDFIC 2 receive odevixibat. For those who received odevixibat in PEDFIC 1, mean serum bile acid levels at PEDFIC 1 baseline were 226 μmol/L in patients with PFIC1 and 263 μmol/L in patients with PFIC2; for those who received placebo in PEDFIC 1, patients with PFIC1 and PFIC2 had mean baseline serum bile acid levels of 200 μmol/L and 263 μmol/L, respectively. The proportion of positive pruritus assessments from Weeks 0–24 during PEDFIC 1 was 61% in patients with PFIC1 and 51% in patients with PFIC2 among those who received odevixibat and 22% and 31%, respectively, among those who received placebo.

MYO5B, myosin 5B; NA, not applicable; PFIC, progressive familial intrahepatic cholestasis.

^†Represents Weeks 0–4 for patients with PFIC1 and PFIC2 and 0–12 for patients with PFIC3 and MYO5B deficiency.

^‡Data are only available for these patient types in cohort 2, per study eligibility criteria for PEDFIC 1 and PEDFIC 2.