Will we see the world's first approved NASH drug in 2020?

Non-alcoholic fatty liver disease
(Nephron, Wikimedia Commons)

Nonalcoholic steatohepatitis (NASH) has been among the closely watched indications in biotech since January 2014, when Intercept Pharmaceuticals lit a fire under the field with midphase data that sent its share price up 500%. Now, Intercept is on the cusp of delivering on the promise that caused investors to pile into the stock.

On April 22, an advisory committee is set to assess the merits of the data on obeticholic acid (OCA), teeing the FDA up to decide whether to make Intercept’s drug the first treatment approved in NASH. Genfit’s elafibranor is following close behind, with phase 3 results scheduled for the first quarter of 2020. If the FDA authorizes either drug, it will mark the start of a new phase in the intense scrap for the blockbuster NASH market.

However, it may take time for the NASH market to live up to the blockbuster forecasts. Neither OCA nor elafibranor has entirely convinced in clinical trials, with Intercept’s drug dogged by a troublesome side effect profile and Genfit’s rival failing a phase 2 study. The data could deter physicians and payers from backing the first NASH drugs, potentially limiting use to the sickest NASH patients.

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Another headwind relates to how NASH is diagnosed. Today, a liver biopsy is needed to make a definitive diagnosis. It is unclear whether payers, physicians and patients will see the costly, invasive procedure as a price worth paying to get the moderate efficacy shown by the leading NASH drugs. In the longer term, noninvasive tests and new NASH drugs and combinations may tip the balance in favor of greater use of pharmacological interventions.

Yet, despite the headwinds, Intercept will need to hit the ground running to live up to expectations. Analysts have tipped OCA to deliver blockbuster sales, with some forecasts made during its clinical development running as high as $8 billion.

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