(Reuters) -The U.S. health regulator’s staff reviewers on Wednesday raised various safety concerns from using Intercept Pharmaceuticals’ treatment for a type of fatty liver disease, dragging the drugmaker’s shares 27% lower in early trade.
The U.S. Food and Drug Administration’s (FDA) briefing document highlighted increased risk of diabetes and liver injury from using the obeticholic acid (OCA) oral tablets for the treatment of nonalcoholic steatohepatitis (NASH).
“During the course of our review, FDA identified modest benefits and serious risks of OCA for treatment of NASH,” the staff said in the document and listed increased risk of sludge formation in the gall bladder and imbalance of certain lipids.
It said the 25 mg dose pills showed superiority compared to placebo in terms of reduction in liver scarring in NASH patients after 18 months, but added that there was uncertainty on how the surrogate endpoint of the ‘biomarker’ might translate into meaningful clinical outcomes.
The staff asked the regulator’s panel of outside advisors, who are meeting on Friday, to consider whether the modest benefits from using the drugs for NASH treatment outweigh the serious risks associated with it.The panel’s meeting on Friday is the first such meeting for a NASH treatment.
With no approved treatments for the disease yet, several drugmakers including Novo Nordisk and other smaller companies Madrigal Pharmaceuticals Inc and Akero Therapeutics Inc are racing to enter what is expected to be a multibillion dollar U.S. market.
In 2016, OCA was approved for treatment of primary biliary cholangitis (PBC), a chronic liver disease in which the small bile ducts become injured and inflamed and are eventually destroyed.
It is sold under the brand Ocaliva for PBC. However, due to risks of serious liver injury, FDA restricted its usage in 2021 to primary biliary cholangitis patients with advanced cirrhosis, or severe liver scarring.
(Reporting by Sriparna Roy and Leroy Leo in Bengaluru; Editing by Rashmi Aich)