US FDA panel votes against approval of Intercept fatty liver drug, cites safety issues (updated)

Intercept Pharmaceuticals

19/05/2023 21:59

(Adds vote on safety data in paragraph 3, panelist quote in
paragraph 5, background)
    By Leroy  Leo and Sriparna  Roy
       May 19 (Reuters) - A panel of advisers to the U.S. Food
and Drug Administration, wary of the safety of Intercept
Pharmaceuticals'  ICPT.O  oral drug for a type of fatty liver
disease, on Friday recommended holding off on an accelerated
approval of the medicine.
    The panel of outside experts voted 15-to-1 against the 
approval for obeticholic acid (OCA) based on surrogate biomarker
data suggesting it was likely to benefit patients with NASH
(non-alcoholic steatohepatitis) and fibrosis, or scarring, of
the liver.
    The panel by a wide 12-to-2 margin voted that the benefits
of Intercept's drug did not outweigh the risks in NASH patients
with fibrosis based on current data. Two members abstained.
    NASH is a huge unmet need with no approved drugs after
numerous clinical failures by several drugmakers. The
progressive fatty liver disease affects about 5% of adults in
the U.S., according to American Liver Foundation.
    "If you're talking about millions of people with NASH who
could go on this drug, 1-in-1000 could get severe DILI
(drug‐induced liver injury), you're talking about a new epidemic
of liver disease as an adverse effect of a drug," said panel
member James Floyd, an associate professor at University of
Washington.
    The condition is the fastest-growing cause of liver
transplants in developed countries, with obesity and diabetes
increasing the risk of NASH.
    Intercept was previously turned down by the FDA in 2020,
with the agency determining that the drug's predicted
effectiveness failed to outweigh potential risks. 
    The drugmaker resubmitted its application last July, with
additional analysis on its effectiveness and safety, but the FDA
still appeared unconvinced. 
    "The FDA initially concluded that the OCA was associated
with an unfavorable benefit/risk profile, and in the
resubmission our assessment of efficacy has remained unchanged,"
Ruby Mehta, FDA's medical team leader for liver diseases said
during Friday's meeting.
    The FDA usually follows the recommendations of its advisory
panels but is not obligated to do so. It has been concerned
about the drug's safety due to a signal of an increased risk of
diabetes and liver injury, among other adverse effects.
    A decision from FDA is expected by June 22. 

 (Reporting by Sriparna Roy and Leroy Leo in Bengaluru; Editing
by Bill Berkrot)
 ((Sriparna.Roy@thomsonreuters.com;))