EMA Recommends EU Marketing Authorization for Bylvay

The European Medicines Agency (EMA) has issued a recommendation to grant the European Union (EU) marketing authorization application for Bylvay (odevixibat) as a treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged six months and older.

PFIC is a rare liver disease that is life-threatening, with approximately only half of the children suffering from the disease surviving past the age of 10 years old. As a result of the limited treatment options available for PFIC patients, there is a high unmet need for therapies to treat this disease.

EMA’s Committee for Medicinal Products for Human Use (CHMP) has based its positive recommendation on the results of a Phase III study, in which the efficacy and safety of Bylvay in children with PFIC were assessed. The clinical trial results demonstrated that the therapy, which selectively inhibits the ileal bile acid transporter, was effective at significantly reducing the serum bile acids and, in patients treated with odevixibat, a significant reduction in pruritus was seen.

The positive results demonstrated with Bylvay were maintained in an ongoing, long-term open label; however, more data are required to confirm if the therapy delays disease progression and the requirement for liver transplantation. Therefore, CHMP has requested a registry-based efficacy study as a follow-up.

Furthermore, as a result of the rarity of PFIC, CHMP has concurred that comprehensive data on the efficacy of the therapy under normal conditions of use will not be plausible. As such, CHMP has recommended that the marketing authorization be granted under exceptional circumstances.

Source: EMA