Albireo Pharma, Inc. announced that the UK Medicines and Healthcare Products Regulatory Agency (MHRA) has granted marketing authorization for Bylvay (odevixibat) for the treatment of all types of progressive familial intrahepatic cholestasis (PFIC). Bylvay is a potent, non-systemic ileal bile acid transport inhibitor (IBATi), administered as a once-daily capsule or opened and sprinkled onto soft foods and which does not require refrigeration. The MHRA authorization follows the European Commission (EC) authorization of Bylvay in July 2021. The MHRA authorization was based on data from PEDFIC 1 and PEDFIC 2, the largest, global, Phase 3 trials ever conducted in PFIC. In PEDFIC 1, a randomized, double-blind, placebo-controlled study, Bylvay met both its pruritus (p=0.004) and serum bile acid (p=0.003) primary endpoints and was well tolerated with low incidence of drug-related diarrhea/frequent bowel movements (9.5% of treated patients vs. 5.0% of placebo patients). PEDFIC 2, a long-term, open-label Phase 3 extension study, affirmed Bylvay delivered sustained reductions in serum bile acid as well as improvements in pruritus assessments, growth and markers of liver function in patients treated up to 48 weeks in an interim analysis. Across both studies, Bylvay was well tolerated with diarrhea/frequent stools being the most common treatment-related gastrointestinal adverse events. There were no serious treatment-related adverse events reported in any clinical study with Bylvay. Albireo has launched Bylvay in the U.S. and is working to commercialize Bylvay in Europe. Bylvay is currently being evaluated by NICE under the Highly Specialised Technologies (HST) pathway, and Albireo is working closely with NICE and NHS England to ensure access for patients in England and Wales as quickly as possible. The Company is also actively engaging with the Scottish Medicines Consortium (SMC). Albireo has developed a compelling value package with the PEDFIC gold standard Phase 3 data, natural history information and data from a recent study reflecting the burden of PFIC on caregivers and families. Bylvay is currently being evaluated in the ongoing PEDFIC 2 open-label trial in patients with PFIC, ASSERT Phase 3 study for Alagille syndrome and in the BOLD Phase 3 study for patients with biliary atresia. The ASSERT and BOLD studies remain on track to report topline data in 2022 and 2024 respectively.