Albireo Pharma, Inc. announced European Commission authorization of Bylvay (odevixibat), the first drug approved for the treatment of all subtypes of progressive familial intrahepatic cholestasis (PFIC). Bylvay is a potent, non-systemic ileal bile acid transport inhibitor (IBATi), which does not require refrigeration and is easily administered as a once-daily capsule or opened and sprinkled onto soft foods. PFIC is a rare and devastating disorder affecting young children that causes progressive, life-threatening liver disease. In many cases, PFIC leads to cirrhosis and liver failure within the first 10 years of life. The most prominent and problematic ongoing manifestation of PFIC is pruritus, or intense itching, which often results in a severely diminished quality of life. Until now, there have been no approved drugs for PFIC; only surgical options that include biliary diversion surgery (BDS) and liver transplantation, and without them most PFIC patients do not survive past the age of 30. Of the estimated 100,000 patients with cholestatic liver disease worldwide without an approved drug treatment, there are approximately 15,000 PFIC patients, excluding China and India. The EC 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 very 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 other 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 plans to directly commercialize Bylvay in the European Union (EU) and is prepared for a global launch with commercial, market access and medical affairs personnel already on the ground. This includes Germany, which has the largest EU market potential, with launch planned for September 2021, following price listing. Pricing and reimbursement dossiers have been submitted to many member states to provide access to Bylvay treatment as soon as possible. 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. The Company also anticipates an upcoming regulatory decision by the U.S. FDA on Bylvay for the treatment of pruritus in patients with PFIC. The FDA has granted a Priority Review for the NDA and has set an action date of July 20, 2021 under the Prescription Drug User Fee Act (PDUFA). Albireo is also studying the use of Bylvay in other rare pediatric cholestatic diseases with the BOLD Phase 3 clinical trial in patients with biliary atresia and the ASSERT Phase 3 clinical trial in Alagille syndrome. Topline data from the ASSERT trial is expected in 2022, and data from the BOLD trial is expected in 2024. Bylvay is the first drug treatment approved in the world for the treatment of all subtypes of progressive familial intrahepatic cholestasis (PFIC). A potent, once-daily, non-systemic ileal bile acid transport inhibitor, Bylvay acts locally in the small intestine. Bylvay does not require refrigeration and can be taken as a capsule for older children, or opened and sprinkled onto food, which are factors of key importance for adherence in a pediatric patient population. Bylvay has an Orphan Designation for the treatment of PFIC. The EMA’s Pediatric Committee has agreed to Albireo’s Bylvay Pediatric Investigation Plans for PFIC, biliary atresia, and Alagille syndrome. In addition to PFIC, Bylvay has Orphan Designations for the treatment of Alagille syndrome, biliary atresia and primary biliary cholangitis. Bylvay is available as capsules. The recommended dose is 40 microgram per kilogram body weight. The capsules should be taken once a day in the morning. They can be taken whole, or they can be opened and sprinkled on food. If the clinical response is inadequate after three months, the dose may be increased up to 120 microgram per kilogram body weight. The medicine can only be obtained with a prescription and treatment should be started and supervised by a doctor, who has experience in the management of PFIC.