The Food and Drug Administration (FDA) has approved Bylvay^® (odevixibat) for the treatment of cholestatic pruritus in patients 12 months of age and older with Alagille syndrome (ALGS).

Bylvay is an ileal bile acid transporter (IBAT) inhibitor. Although the mechanism by which odevixibat improves pruritus in ALGS 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.

The approval was supported by data from the phase 3 ASSERT study (ClinicalTrials.gov Identifier: NCT04674761), which included 52 patients aged 6 months to 15 years with a confirmed diagnosis of ALGS and presence of pruritus at baseline. Patients were randomly assigned to receive odevixibat 120mcg/kg (n=35) or placebo (n=17) orally once daily for 24 weeks.

Results showed that treatment with odevixibat met the primary endpoint demonstrating a statistically significant reduction in pruritus at month 6, as measured by the observer-reported outcome scratching score, compared with placebo (mean difference vs placebo, -0.9 [95% CI, -1.4, -0.3]; P =.002). The most common adverse reactions reported with treatment included diarrhea, abdominal pain, hematoma, and decreased weight.

“Physicians urgently need more options to treat patients with Alagille syndrome and this approval from the US FDA spotlights the robustness of the phase 3 ASSERT clinical study results,” said Nadia Ovchinsky, MD, Chief of the Division of Gastroenterology and Hepatology, Hassenfeld Children’s Hospital at NYU Langone and ASSERT Principal Investigator. “The ASSERT study showed that Bylvay reduced pruritus associated with ALGS, which is so common among this patient population and one of the leading indications for a liver transplant.”

Bylvay Approved for Cholestatic Pruritus Due to Alagille Syndrome

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