Alfasigma to License GSK Drug for Rare Liver Disease

Rare Daily Staff

Drug giant GSK has agreed to license its experimental drug linerixibat to Italian pharmaceutical company Alfasigma, granting the firm exclusive worldwide rights to develop, manufacture, and commercialize the medicine for patients the rare liver disease primary biliary cholangitis.

Under the deal, GSK will receive a $300 million upfront payment and an additional $100 million upon expected approval by the U.S. Food and Drug Administration later this month. The company may also earn $20 million for approvals in the European Union and United Kingdom and up to $270 million in sales-based milestone payments, plus royalties on global sales.

Primary biliary cholangitis (PBC) is a chronic autoimmune disorder in which the bile ducts in the liver are gradually destroyed, preventing normal bile flow. When bile acids build up in the body, many patients experience cholestatic pruritus—an intense, internal itching that cannot be relieved by scratching. The condition can disrupt sleep, worsen fatigue, and severely affect quality of life. In the United States, studies suggest that as many as one-third of patients with significant itching do not receive any treatment.

Linerixibat is an oral drug that blocks the ileal bile acid transporter (IBAT), a mechanism involved in reabsorbing bile acids in the intestine. By reducing the accumulation of these acids, the drug aims to ease the debilitating itch associated with PBC. In a phase 3 clinical trial linerixibat significantly improved itching and itch-related sleep disturbance compared with placebo, with a safety profile consistent with previous studies. The drug has received orphan drug designation in the United States, European Union, and Japan, and is under regulatory review in multiple markets. It is not yet approved anywhere in the world.

“Alfasigma is committed to advancing rare and specialty care by developing and delivering innovative solutions that address some of the most complex healthcare challenges,” said Francesco Balestrieri, CEO of Alfasigma. “With our deep hepatology expertise and strong global footprint, we are uniquely positioned to lead the worldwide commercialization of linerixibat. This agreement underscores our strategic focus on bringing meaningful new treatments to patients and improving outcomes for communities around the world.”

Photo: Francesco Balestrieri, CEO of Alfasigma