19 deaths from liver drug causes Intercept shares to tumble

Intercept Pharmaceuticals had sparked major concerns over its drug, Ocaliva, after it revealed that 10 people had died as a result of its treatment – that concerned escalated as the FDA announced 19 people in total had died.

In the initial letter sent out to health care providers (HCPs), Intercept warned of patient deaths that had resulted from patients being dosed more frequently than recommended with the treatment.

Ocaliva is a farnesoid X receptor agonist approved for the treatment of primary biliary cholangitis, a rare condition of the liver, wherein the bile ducts are inflamed and can potentially become irreparably damaged.

The FDA revealed that 19 deaths and 11 cases of serious liver injury had been associated with the use of the drug but Intercept has pinned the blame firmly on the HCPs involved. The FDA seemed to corroborate this stance by referring to in its missive by stating that "the liver disease medicine Ocaliva is being incorrectly dosed in some patients with moderate to severe decreases in liver function".

Further than this, the FDA’s message carries strict dosing regimens, including taking the time to highlight that the drug should be used, initially, once weekly rather than daily – as in other forms of treatment for the condition.

Whether this may be enough to stop the FDA slapping a black box warning on the label remains to be seen. The number of deaths will be particularly worrying, but the risks of the drug, when given in higher doses, are already a part of the current label.

In either case, the release of the news was enough to cause investors to panic and sent the value of its stock down by close to 25%. The product is the company's only approved treatment.

Ocaliva had been mooted as a potential blockbuster treatment, as it seeks to expand its indication into the lucrative NASH market. However, such worrying safety signs, may be enough to tank the treatment’s hope of gaining strong sales in a highly competitive market.