First Potential Drug for NASH Granted Fast Track Designation

Shire’s investigational SHP626 (volixibat) treatment for nonalcoholic steatohepatitis (NASH) with liver fibrosis has just received fast track designation from the FDA.

The treatment is being developed as a once-daily, orally-administered inhibitor of the apical sodium dependent bile acid transporter, a protein primarily responsible for recycling bile acids from the intestine to the liver. Importantly, there are no currently approved drugs for NASH, a serious, chronic liver disease.

“Shire’s development plan for SHP626 is designed to address the unmet need in the treatment of adult patients who have NASH with liver fibrosis,” said Philip J. Vickers, PhD, head of R&D at Shire. “This fast track designation is further recognition of the critical need to develop new, effective therapeutic options for patients with this serious condition.”

The designation for NASH was supported by preclinical and phase 1 studies that assessed the safety and tolerability of the drug compared with placebo in healthy volunteers, as well as in overweight and obese patients. Shire is also set to start a phrase 2 trial, randomized, placebo-controlled, double-blind study to evaluate the safety, tolerability, and efficacy of 3 doses of SHP626 given to adult patients over the course of 48 weeks.

Adverse events that occurred in phase 1 trials were predominantly diarrhea that wasn’t considered serious. One serious adverse event that led to treatment discontinuation was alanine aminotransferase elevation.