- The Food and Drug Administration on Wednesday restricted the use of Intercept Pharmaceuticals' liver disease drug Ocaliva after an investigation linked it to severe injuries including liver failure in certain patients.
- In most cases, the benefits of Ocaliva outweigh the risks for patients with primary biliary cholangitis, the FDA said. But the agency announced Wednesday that doctors should not prescribe the medicine for patients with advanced cirrhosis, or permanent scarring.
- The new contraindication follows a safety warning added in 2018 to caution physicians about overdosing patients with Ocaliva and recommend they engage in closer monitoring. In the latest update, the FDA said it's connected the drug to 25 cases of injury leading to liver decompensation or liver failure in patients with cirrhosis.
The FDA action won't have an immediate impact on sales of Ocaliva because the majority of patients taking the drug don't have advanced cirrhosis, according to Intercept. The company prepared investors for the move earlier this month when it released first-quarter financial results.
Still, the additional warning to doctors may affect future prescriptions, SVB Leerink analyst Thomas J. Smith wrote in a note to investors.
Any increased concern among doctors would put Intercept at a disadvantage should competitors reach the market. Both CymaBay Therapeutics and Genfit are developing treatments for primary biliary cholangitis, a rare disease that affects the liver ducts that carry digestion-aiding bile. Those companies are likely to report Phase 3 results from their drugs by early 2023, Smith said.
Intercept hoped to bring Ocaliva into a much larger market for the liver disease NASH, or nonalcoholic steatohepatitis. But the company has hit major setbacks in that quest.
In June 2020 — the month after the FDA began investigating safety reports about Ocaliva in primary biliary cholangitis patients — agency officials rejected an expanded use of the drug for NASH, saying they weren't convinced the benefits outweighed the risks for that patient population. Since then, Intercept has been gathering new data and trying to conserve its cash, laying off a quarter of its staff as a result.
Earlier this month, the company said it's had multiple interactions with the FDA and plans to submit research on exposure to Ocaliva in twice as many patients as the original submission. Analysts at Jefferies now expect a re-filing in 2022.
Sales of Ocaliva rose 12% to $82 million in the first quarter this year, according to Intercept. The company forecast sales of between $325 million and $340 million for the year.