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NASH was once the next big thing for big drug companies; a newly defined disorder that requires chronic treatment that affects millions of people in developed countries. Dozens of companies are developing drugs for the disease, which can cause life-threatening conditions like decompensated cirrhosis or liver cancer.
But the search for the first great NASH drug has run into some obstacles. In May, a French company called Genfit (ticker: GNFT), which had one of the most advanced NASH programs, announced that a phase 3 trial of its NASH drug Elafinbranor had failed to achieve its primary goal.
Now, another setback is the drop in shares of Intercept Pharmaceuticals (ICPT), which analysts had hoped to unveil the first NASH drug sometime this year. Intercept had filed a request for expedited approval with the Food and Drug Administration for its obeticholic acid as a treatment for NASH. On Monday, the company said the FDA had sent him a letter saying the expected benefit of the drug “does not sufficiently outweigh the potential risks.”
The company said the agency had requested additional analysis of an ongoing study. Intercept shares fell 39% on Monday. In a call to investors Monday morning, Intercept CEO Mark Pruzanski called the FDA action “completely unexpected and disappointing.”
“At no point during the review, the FDA reported that OCA was not expeditable,” said Pruzanski.
Questions about NASH medications have arisen for months. In December, Barron’s He highlighted those concerns, arguing that investors should be skeptical about the field.
Intercept’s request for OCA was based on provisional data from a study that found that liver scarring improved, and other NASH parameters did not worsen, in 23.1% of patients receiving a higher dose of the drug, compared to 11.9% of patients on placebo. arm of the study.
In an interview in December, Pruzanski said Barron’s that the most important metric was fibrosis stabilization and that 40% of patients in the high-dose group improved in a full stage of fibrosis.
The drug also raises levels of so-called bad cholesterol, which could be a concern since patients who are likely to take the drug to treat NASH are also prone to heart disease. In the December interview, an Intercept executive told him Barron’s that the risk could be managed with other dugs.
Intercept shares have fallen 61.8% so far this year.
Write to Josh Nathan-Kazis at [email protected]
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