Intercept Pharmaceuticals has submitted a New Drug Application (NDA) to the Food and Drug Administration (FDA), with a request for Priority Review, for obeticholic acid (OCA) for the treatment of patients with fibrosis due to nonalcoholic steatohepatitis (NASH).

OCA is an agonist of the Farnesoid X receptor (FXR), which has demonstrated to be a key regulator of bile acid, metabolic, inflammatory, and fibrotic pathways.

The NDA submission is based on data from the phase 3, double-blind, placebo-controlled, multicenter, REGENERATE study that evaluated the safety and efficacy of OCA in 2480 patients with liver fibrosis due to NASH (histological evidence of fibrosis stage 2 or stage 3). Patients were randomized 1:1:1 to receive OCA 10mg, 25mg, or placebo once daily for 18 months. The primary end points were the proportion of patients achieving at least 1 stage of liver fibrosis improvement with no worsening of NASH or the proportion of patients achieving NASH resolution with no worsening of liver fibrosis.

Results demonstrated that treatment with OCA 25mg once daily met the primary end point of fibrosis improvement, achieving ≥1 stage of liver fibrosis improvement with no worsening of NASH at the planned 18-month analysis (P=.0002 vs placebo). Both doses of OCA also showed NASH resolution with no worsening of liver fibrosis, but failed to reach statistical significance. Regarding safety, the most common adverse reaction reported was dose-related pruritus.

The Company is also conducting a phase 3 study (REVERSE) to evaluate whether OCA can lead to histological improvement in fibrosis with no worsening of NASH in patients with compensated cirrhosis due to NASH.

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“Our submission of the first NDA for the treatment of fibrosis due to NASH is a very important milestone for the field and the culmination of more than a decade of hard work,” said Mark Pruzanski, MD, President and CEO of Intercept. “We look forward to continuing to work with the FDA through the NDA review period and believe that, if approved, OCA has the potential to become an essential treatment for people living with advanced fibrosis due to NASH.”

Obeticholic acid (Ocaliva) is currently approved for the treatment of primary biliary cholangitis in combination with ursodeoxycholic acid (UDCA) in adults with inadequate response to UDCA, or as monotherapy in adults unable to tolerate UDCA.

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