Initiation of Global Phase 3 Trial of Alagille Syndrome Treatment

Albireo has recently initiated their Phase 3 ASSERT trial, according to GlobeNewswire. This study will evaluate odevixibat as a treatment for Alagille syndrome. This therapy fits well with Albireo’s mission, which is developing treatments for rare liver diseases. In fact, odevixibat has also been indicated for PFIC and biliary atresia.

About Alagille Syndrome

Alagille syndrome is a rare, genetic disorder that impacts multiple systems throughout the body, including the liver, kidneys, skeleton, eyes, and heart. The effects on these systems can vary widely throughout affected individuals, but liver disease and heart abnormalities are typically seen. Other symptoms include failure to thrive, jaundice, abnormalities of the back bone, distinctive facial features, pruritus, heart defects, cholestasis, heart murmurs, and pale and loose stools. These symptoms are the result of a mutated JAG1 or NOTCH2 gene. Both are usually inherited in an autosomal dominant pattern, but they can be sporadic mutations as well. There is currently no cure for Alagille syndrome; treatment is symptomatic and supportive.

About the Trial

The ASSERT trial is double-blind, placebo-controlled, and randomized in an effort to investigate the safety and efficacy of 120 µg/kg/day of odevixibat for 24 weeks. Researchers will focus on its affect on the severe itching that characterizes Alagille syndrome, or pruritus. They plan to enroll 45 patients under the age of 18 across 35 sites in the US, Middle East, Europe, and Asia Pacific.

The FDA and EMA have stated that this trial is sufficient for regulatory filings if it is successful. This statement comes after Albireo submitted a New Drug Application to the FDA and Marketing Authorization Application to the EMA for odevixibat as a treatment for PFIC.

Hopefully this trial demonstrates positive results, as patients with Alagille syndrome currently face an unmet medical need.

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