One main study has shown that Bylvay is effective at reducing the amount of bile acid in the blood of patients with PFIC. Bylvay was also effective at reducing signs and symptoms of PFIC such as itching. Because PFIC is a very rare disease, the study was small but the short-term data available indicated that Bylvay could delay disease progression and the need for surgery and/or liver transplantation. The side effects seen to date are considered manageable. Given the seriousness of the condition and the lack of existing treatments, the European Medicines Agency decided that Bylvay’s benefits are greater than its risk and it can be authorised for use in the EU.

Bylvay has been authorised under ‘exceptional circumstances’. This means that, because the indication is encountered so rarely, it has not been possible to obtain full information about the medicine. Every year, the European Medicines Agency will review any new information that may become available and this summary will be updated as necessary.