BYLVAY

What it is

BYLVAY contains odevixibat hard capsules available in strengths of 200 micrograms, 400 micrograms, 600 micrograms, or 1200 micrograms. Odevixibat is a reversible and selective inhibitor of the ileal bile acid transporter (IBAT). Odevixibat acts locally in the distal ileum to decrease the reuptake of bile acids and increase the clearance of bile acids through the colon, reducing the concentration of bile acids in the serum.

Approved indications

— Treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older.

Dosing overview

The recommended dose of odevixibat is 40 micrograms/kg administered orally once daily in the morning. Odevixibat can be taken with or without food. If an adequate clinical response has not been achieved after 3 months of continuous therapy, the dose may be increased to 120 micrograms/kg/day. Alternative treatment should be considered in patients for whom no treatment benefit can be established following 6 months of continuous daily treatment with odevixibat.

Key safety warnings

Diarrhoea has been reported as a common adverse reaction when taking odevixibat and may lead to dehydration. Patients should be monitored regularly to ensure adequate hydration during episodes of diarrhoea. Assessment of fat-soluble vitamin levels (Vitamins A, D, E) and international normalised ratio (INR) are recommended for all patients prior to initiating BYLVAY, with monitoring per standard clinical practice. In clinical trials, decreased levels of fat-soluble vitamins were observed in some patients receiving odevixibat. Levels of fat-soluble vitamins should be monitored. In clinical trials, increased levels in liver function tests were observed in some patients receiving odevixibat. Assessment of liver function tests is recommended for patients prior to initiating BYLVAY, with monitoring per standard clinical practice.

Contraindications

Hypersensitivity to the active substance or to any of the excipients.

PBS listing

BYLVAY capsules are listed on the PBS in four strengths: 200 micrograms, 400 micrograms, 600 micrograms, and 1.2 mg, each with 6 PBS items under authority required restriction. Ex-manufacturer prices are A$5580.00, A$11160.00, A$16740.00, and A$33480.00 respectively, with the latest schedule dated 2026-05-01.

Regulatory history

BYLVAY received TGA approval on 24 March 2024 for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older. All four strengths were registered on the ARTG on 26 March 2025. The PBAC initially did not recommend BYLVAY in July 2024, and the application was deferred in March 2025. BYLVAY was recommended by PBAC in July 2025 as superior to standard of care, following a resubmission that addressed outstanding issues including price reduction and revised financial estimates.