MHRA approves Bylvay for progressive familial intrahepatic cholestasis – Albireo Pharma
Albireo Pharma 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). 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 III 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 III 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.