Bylvay is the first and only FDA-approved treatment for all levels of cholestatic pruritus in all PFIC types1,3
PFIC 1
ATP8B1 gene defect (FIC1 protein deficiency)4
PFIC 2
ABCB11 gene defect (BSEP protein deficiency). Only 21% of patients have a BSEP-3 deficiency4,5
PFIC 3
ABCB4 gene defect (MDR3 protein deficiency)4
PFIC 4
TJP2 gene defect (TJP2 and CLDN1 protein dysfunctions)4,6
PFIC 10
(formerly known as PFIC 6) MYO5B gene defect (defects in MYO5B and RAB11A proteins)4,6,7
No matter what type of PFIC you’re treating, consider Bylvay for cholestatic pruritus in your patients1
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
See the full Limitation of Use.
Early and sustained reductions in cholestatic pruritus are possible
BSEP=bile salt export pump; CLDN1=claudin-1; FIC1=familial intrahepatic cholestasis protein 1; MDR3=multidrug resistance protein 3; MYO5B=myosin Vb; PFIC=progressive familial intrahepatic cholestasis; RAB11A=Ras-related protein Rab-11A; TJP2=tight junction protein 2.
References:
- Bylvay Prescribing Information. Boston, MA: Albireo Pharma, Inc.; 2023.
- Özen H, Sokal E, Lacaille F, et al. Efficacy and safety outcomes with odevixibat in children with progressive familial intrahepatic cholestasis due to deficiencies in multidrug resistance protein 3 (PFIC type 3) or myosin 5B (PFIC type 6). Presented at: AASLD: The Liver Meeting 2021; November 12-15, 2021.
- Kamath BM, Stein P, Houwen RHJ, Verkade HJ. Potential of ileal bile acid transporter inhibition as a therapeutic target in Alagille syndrome and progressive familial intrahepatic cholestasis. Liver Int. 2020;40(8):1812-1822.
- Goldberg A, Mack CL. Inherited cholestatic diseases in the era of personalized medicine. Clin Liver Dis (Hoboken). 2020;15(3):105-109.
- van Wessel DBE, Thompson RJ, Gonzales E, et al. Genotype correlates with the natural history of severe bile salt export pump deficiency. J Hepatol. 2020;73(1):84-93.
- Amirneni S, Haep N, Gad MA, Soto-Gutierrez A, Squires JE, Florentino RM. Molecular overview of progressive familial intrahepatic cholestasis. World J Gastroenterol. 2020;26(47):7470-7484.
- Online Mendelian Inheritance in Man® (OMIM®); 2023. Accessed January 17, 2023. https://www.omim.org/clinicalSynopsis/table?
mimNumber=601847,619484,619658,602347,615878,619662,617049,619849,619874,620010,619868,211600&from=PS211600.
- Bylvay Prescribing Information. Boston, MA: Albireo Pharma, Inc.; 2023.
- Özen H, Sokal E, Lacaille F, et al. Efficacy and safety outcomes with odevixibat in children with progressive familial intrahepatic cholestasis due to deficiencies in multidrug resistance protein 3 (PFIC type 3) or myosin 5B (PFIC type 6). Presented at: AASLD: The Liver Meeting 2021; November 12-15, 2021.
- Kamath BM, Stein P, Houwen RHJ, Verkade HJ. Potential
of ileal bile acid transporter inhibition as a therapeutic
target in Alagille syndrome and progressive familial
intrahepatic cholestasis. Liver Int. 2020;40(8):1812-1822. - Goldberg A, Mack CL. Inherited cholestatic diseases in the era of personalized medicine. Clin Liver Dis (Hoboken). 2020;15(3):105-109.
- van Wessel DBE, Thompson RJ, Gonzales E, et al. Genotype correlates with the natural history of severe bile salt export pump deficiency. J Hepatol. 2020;73(1):84-93.
- Amirneni S, Haep N, Gad MA, Soto-Gutierrez A, Squires
JE, Florentino RM. Molecular overview of progressive
familial intrahepatic cholestasis. World J Gastroenterol. 2020;26(47):7470-7484. - Online Mendelian Inheritance in Man® (OMIM®); 2023.
Accessed January 17, 2023. https://www.omim.org/
clinicalSynopsis/table? mimNumber=601847,619484,619658,602347,615878,619662,617049,619849,619874,620010,619868,211600&from=PS211600.