Alagille Syndrome
Progressive Familial Intrahepatic Cholestasis
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Albireo initiates global phase 3 Clinical trial of odevixibat in Alagille syndrome

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Albireo Pharma, Inc, a clinical-stage rare liver disease company developing novel bile acid modulators, today announced the initiation of its global Phase 3 pivotal trial, ASSERT, Alagille Syndrome looking at Safety and Efficacy in a Randomized controlled Trial, which will evaluate odevixibat in patients with Alagille syndrome. Odevixibat is a potent, once-daily, non-systemic ileal bile acid transport inhibitor (IBATi) being investigated for the treatment of rare pediatric cholestatic liver diseases, including progressive familial intrahepatic cholestasis (PFIC), biliary atresia and Alagille syndrome (ALGS). ASSERT is Albireo’s third global trial in rare cholestatic liver conditions and furthers the Company’s efforts to deliver life-changing therapies to children and young adults living with these diseases.

ALGS is a rare multisystem genetic disorder that can affect the liver, heart and other parts of the body. Approximately 95% of patients with the condition present with chronic cholestasis, usually within the first three months of life, and as many as 88% also present with severe, intractable pruritus. Currently, there is no approved drug therapy for the treatment of ALGS.

ASSERT is a gold standard, prospective intervention trial. The double-blind, randomized, placebo-controlled trial is designed to evaluate the safety and efficacy of 120 µg/kg/day odevixibat for 24 weeks in relieving pruritus in patients with ALGS. Secondary endpoints will measure serum bile acid levels and safety and tolerability. Both the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) have agreed on the study design and have indicated that a single study demonstrating safety and efficacy of odevixibat would be sufficient for regulatory filings.  The trial is expected to enroll approximately 45 patients aged 0 to 17 years of age with a genetically confirmed diagnosis of ALGS across 35 sites in North America, Europe, Middle East and Asia Pacific.

“Odevixibat is the first IBAT inhibitor to have demonstrated efficacy and tolerability in a Phase 3 randomized, placebo-controlled trial, and this gives us increased confidence for positive clinical outcomes in Alagille syndrome with ASSERT,”  said Ron Cooper, President and Chief Executive Officer of Albireo. “We are pleased to initiate the ASSERT study within guidance and offer hope to children and young adults around the globe with Alagille syndrome who have no approved therapeutic options today.”

Albireo recently submitted for a New Drug Application (NDA) to the U.S. FDA and a Marketing Authorization Application (MAA) to the EMA seeking approval of odevixibat for the treatment of patients with PFIC. Odevixibat has previously received Fast Track, Rare Pediatric Disease and Orphan Drug Designations in the U.S. In addition to PFIC, odevixibat has Orphan Drug Designations for the treatment of Alagille syndrome, biliary atresia and primary biliary cholangitis. The EMA has granted odevixibat accelerated assessment, Orphan Designation, as well as access to the PRIority MEdicines (PRIME) scheme for the treatment of PFIC. The EMA’s Pediatric Committee has agreed to Albireo’s odevixibat Pediatric Investigation Plans for PFIC and biliary atresia. With U.S. and EU regulatory submissions for odevixibat in PFIC completed, the Company anticipates potential regulatory approvals, issuance of a rare pediatric disease priority review voucher and launch in the second half of 2021.

Odevixibat is also currently being evaluated in the ongoing PEDFIC 2 Phase 3 open-label trial in patients with PFIC, and the BOLD Phase 3 trial in patients with biliary atresia. The Company provides an Expanded Access Program for eligible patients with PFIC in the U.S., Canada, Australia and Europe.

Read the full press release here.