General Information

Bylvay (odevixibat) is a potent, non-systemic ileal bile acid transport inhibitor (IBATi).

Bylavy is specifically indicated for the treatment of pruritus in patients 3 months of age and older with progressive familial intrahepatic cholestasis (PFIC).

Bylavy is supplied as capsules and pellets for oral administration. Bylavy oral pellets are intended for use by patients weighing less than 19.5 kilograms. Bylavy capsules are intended for use by patients weighing 19.5 kilograms or above.

The recommended dosage of Bylavy is 40 mcg/kg once daily in the morning with a meal. If there is no improvement in pruritus after 3 months, the dosage may be increased in 40 mcg/kg increments up to 120 mcg/kg once daily not to exceed a total daily dose of 6 mg. 

Mechanism of Action

Bylvay (odevixibat) is a potent, non-systemic ileal bile acid transport inhibitor (IBATi). It decreases the reabsorption of bile acids (primarily the salt forms) from the terminal ileum. Pruritus is a common symptom in patients with PFIC and the pathophysiology of pruritus in patients with PFIC is not completely understood. Although the complete mechanism by which odevixibat improves pruritus in PFIC patients is unknown, it may involve inhibition of the IBAT, which results in decreased reuptake of bile salts, as observed by a decrease in serum bile acids.

Side Effects

Adverse effects associated with the use of Bylavy may include, but are not limited to, the following:

• liver test abnormalities
• diarrhea
• abdominal pain
• vomiting
• fat-soluble vitamin deficiency

Clinical Trial Results

The approval of Bylvay was supported by data from PEDFIC 1 and PEDFIC 2, the largest, global, Phase 3 trials ever conducted in PFIC. In PEDFIC1, a randomized, double-blind, placebo-controlled study, Bylvay met both its pruritus and serum bile acid primary endpoints and was well tolerated with very low incidence of diarrhea/frequent bowel movements (9.5% of treated patients vs. 5.0% of placebo patients). PEDFIC 2, a long-term, open-label Phase 3 extension study, reaffirmed Bylvay delivered sustained reductions in serum bile acids as well as improvements in pruritus assessments, growth and other markers of liver function in patients treated up to 48 weeks.