The EMA’s CHMP has recommended EU approval of Lynavoy for cholestatic pruritus in adults with primary biliary cholangitis, based on Phase 3 data.

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended approval of Lynavoy (linerixibat) in the European Union for the treatment of cholestatic pruritus in adult patients with primary biliary cholangitis (PBC).

The positive opinion is based on data from the Phase 3 GLISTEN study, which met its primary and key secondary endpoints and showed improvements in cholestatic pruritus and itch-related sleep interference compared with placebo.

The European Commission is expected to make a final decision on the marketing authorisation application in the coming months.

If approved, Lynavoy would be the first ileal bile acid transporter (IBAT) inhibitor available in the EU specifically for the treatment of cholestatic pruritus in adults with PBC.

Primary biliary cholangitis is a rare autoimmune liver disease in which bile flow from the liver is disrupted. The condition can progress to cirrhosis, liver failure and, in some cases, the need for liver transplantation.

Cholestatic pruritus is a common symptom of PBC, affecting up to 89% of people living with the disease. The condition causes an internal itch that cannot be relieved by scratching and can have a significant impact on sleep, fatigue and quality of life.

The GLISTEN Phase 3 study found that treatment with linerixibat resulted in a rapid, significant and sustained improvement in cholestatic pruritus and itch-related sleep interference compared with placebo.

The safety profile of linerixibat was consistent with previous clinical studies and its mechanism of action as an IBAT inhibitor.

Francesco Balestrieri, chief executive officer of Alfasigma, said: “The CHMP’s positive opinion for linerixibat is an important milestone for people living with primary biliary cholangitis who experience cholestatic pruritus, a debilitating symptom that can significantly impact quality of life.”

Cholestatic pruritus can occur at any stage of PBC, including in patients whose disease is otherwise under biochemical control. Despite its impact, the symptom may not always be documented or treated.

Data from the US indicate that itch is frequently missing from medical records, while up to one-third of patients experiencing clinically significant itch do not receive treatment.

Linerixibat is an IBAT inhibitor that works by targeting the ileal bile acid transporter, reducing the circulation of bile acids thought to contribute to cholestatic pruritus.

The treatment has already received approval for use in the US and UK.

Alfasigma acquired worldwide exclusive rights to develop, manufacture and commercialise linerixibat through a licensing agreement with GSK.

The positive CHMP opinion marks the latest regulatory milestone for the treatment as Alfasigma seeks to expand access to linerixibat for people living with PBC-associated cholestatic pruritus.

Final approval by the European Commission would allow the treatment to become available in EU markets, subject to national access and reimbursement decisions.

The recommendation adds a potential new treatment option for patients living with one of the most burdensome symptoms associated with primary biliary cholangitis, a rare liver disease that can have a significant impact on daily life and long-term health.