Albireo Pharma, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted the Company's supplemental New Drug Application (sNDA) and issued a Prescription Drug User Fee Act (PDUFA) action date of June 15, 2023 for a second Bylvay (odevixibat) indication for patients with Alagille syndrome (ALGS). As defined by the FDA, the Priority Review timeline is 6 months and the agency will direct overall attention and resources to the evaluation of applications for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. With completed submissions for Bylvay in the U.S. and EU for use in patients with ALGS, the company anticipates approvals in mid-2023.

ASSERT Phase 3 Clinical Trial Data: ASSERT is a gold standard, prospective intervention trial with 32 sites across North America, Europe, Middle East, and Asia Pacific. The double-blind, randomized, placebo-controlled trial was designed to evaluate the safety and efficacy of 120 µg /kg/day Bylvay for 24 weeks in relieving pruritus in patients with ALGS. Key secondary endpoints measure serum bile acid levels and safety and tolerability.

The trial enrolled patients aged 0 to 17 years of age with a genetically confirmed diagnosis of ALGS. In the primary analysis, the study met the primary endpoint showing statistically significant reduction in pruritus as measured by the PRUCISION Observer-Reported Outcome scratching score (0-4 point scale), from baseline at month 6 (weeks 21 to 24), compared to the placebo arm (p=0.002). Over 90% of patients were pruritus responders during the study, as defined as at least a 1-point drop at any time point.

The study also met the key secondary endpoint showing a statistically significant reduction in serum bile acid concentration from baseline to the average of weeks 20 and 24 (compared to the placebo arm p=0.001). Statistically significant improvements in multiple sleep parameters were observed as early as week 1-4 compared to patients on placebo with continued improvement through week 24. In the study, there were no patient discontinuations.

Bylvay was well tolerated, with an overall adverse event incidence similar to placebo and a low incidence of drug-related diarrhea (11.4% vs. 5.9% placebo).