Albireo’s Bylvay approved in EU and US for rare paediatric liver disease

Albireo Pharma’s Bylvay has received approval on both sides of the Atlantic this week, scoring authorisation in the EU and US for the treatment of all subtypes of progressive familial intrahepatic cholestasis (PFIC).

PFIC is a rare and progressive liver disease affecting young children which can lead to cirrhosis and liver failure within the first ten years of life.

The most common symptoms of PFIC is pruritus (intense itching) which can often result in a severely diminished quality of life.

Bylvay (odevixibat) is the first drug to be approved for PFIC – prior to its approval, only surgical options including biliary diversion surgery (BDS) and liver transplantation have been available to these patients.

The approvals are supported by data from the Phase III PEDFIC 1 and PEDFIC 2 trials. In the PEDFIC 1 trial, Bylvay met both its pruritus and serum bile acid primary endpoints and was found to be well tolerated.

Meanwhile, in the PEDFIC 2 trial – a long-term extension study – 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.

Bylvay is a non-systemic ileal bile acid transport inhibitor (IBATi) which is administered as a once-daily capsule or opened and sprinkled onto soft foods.

“Until now invasive surgery was the only approved treatment option. With the approval of Bylvay, parents may find hope in having a less invasive treatment option available,” said Emily Ventura, leader of the PFIC Advocacy and Resource Network.

“As a community, we experience extreme challenges and diminished quality of life for children and families with PFIC. Managing the symptoms can be extremely difficult – the burden is unimaginable with our kids suffering physically, emotionally and developmentally,” she added.