Ipsen's IBAT Inhibitor Bylvay Receives FDA Approval for New Indication in Pediatric Alagille Syndrome

On June 13, Ipsen announced on its official website that the FDA had approved the listing application for a new indication of Bylvay (odevixibat) for the treatment of cholestatic pruritus in patients with Alagille syndrome aged 12 months and above.

Alagille Syndrome (ALGS) is an autosomal dominant genetic disorder and also one of the important causes of chronic cholestatic liver disease in infancy. ALGS mainly manifests as intrahepatic bile duct paucity and cholestasis. Relevant studies have shown that ileal bile acid transporter (IBAT)/apical sodium-dependent bile acid transporter (ASBT) inhibitors demonstrate good therapeutic effects for ALGS.

Bylvay is a potent, non-systemic ileal bile acid transporter inhibitor (IBATi) with minimal systemic exposure, acting locally only within the intestine. In July 2021, Bylvay received FDA approval, becoming the first drug for the treatment of pruritus in all subtypes of progressive familial intrahepatic cholestasis (PFIC).

ASSERT Study Design (Source: Albireo Official Website)

The approval was based on positive data from the global, double-blind, randomized, placebo-controlled Phase III ASSERT study. The study aimed to evaluate the safety and efficacy of Bylvay in patients with ALGS, with the primary endpoint being the change in pruritus levels from baseline to month 6 (weeks 21-24); key secondary endpoints included changes in the average serum bile acid levels from baseline to month 6 (weeks 21-24).

The study results showed that, compared with the placebo group, the study achieved the primary endpoint, which was a statistically significant reduction (p=0.002) in pruritus symptoms measured by the PRUCISION observer-reported itch score (0-4 scale) from baseline to 6 months (weeks 21-24). The study also achieved key secondary endpoints, showing a statistically significant decrease (p=0.001) in serum bile acid concentration from baseline to the average values at weeks 20 and 24 compared with the placebo group. As early as weeks 1-4, multiple sleep parameters showed statistically significant improvements compared with patients in the placebo group, with continued improvement up to week 24. In terms of safety, Bylvay treatment was well-tolerated, with an overall adverse event rate similar to that of the placebo.

Bylvay was originally developed by Albireo Pharma. In March 2023, Ipsen completed the acquisition of this company to expand its rare disease treatment portfolio.

In addition to PFIC and Alagille syndrome, Albireo is also conducting the Phase III BOLD study for Bylvay in the indication of biliary atresia (BA). Biliary atresia is the most common pediatric cholestatic liver disease, and there are currently no approved drugs. Albireo continues to recruit patients for the BOLD study, which is the first and only clinical trial of an IBATi for the indication of biliary atresia (BA). The study is expected to provide top-line data in 2024.

Copyright © 2023 PHARMCUBE. All Rights Reserved.

Welcome to forward, share, and reasonably cite. When citing, please clearly indicate the source of the article; if you need to reprint, please leave a message to the WeChat Official Account or send a message, and include the name and ID of the official account.

Disclaimer: The information in this WeChat article is for general reference only and should not be directly used as decision-making content. PharmaCube assumes no responsibility for any loss incurred by any party due to the use of the content herein.

Exciting Preview

Online Live Streaming & Offline Salon