FDA grants fast track for Shire’s NASH investigational treatment

Shire Plc has announced today that the United States Food and Drug Administration (FDA) has granted Fast Track designation for SHP626 (volixibat) for an investigational treatment of adults with nonalcoholic steatohepatitis (NASH) with liver fibrosis.

Global biotechnology company, Shire, is developing SHP626 as a once daily, orally-administered inhibitor of the apical sodium dependent bile acid transporter (ASBT) – a protein that is primarily responsible for recycling bile acids from the intestine to the liver. NASH is a serious, chronic liver disease that can be severe and lead to fibrosis, cirrhosis, liver failure and liver cancer. There are currently no approved drugs.

“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, Ph.D., Head of R&D, Shire.

Promising preliminary results for NASH

 The FDA Fast Track Designation for SHP626 in NASH was supported by preclinical and Phase 1 studies. The FDA’s Fast Track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. However, it does not guarantee that the FDA will ultimately approve SHP626 for NASH or the timing of any such approval.

 Shire will initiate its Phase 2 trial with SHP626 as a randomised, placebo-controlled, double-blind study to evaluate the safety, tolerability and efficacy of three doses of volixibat over a 48-week period in adult patients with NASH. The Phase 2 study will be conducted in the U.S., Canada and the United Kingdom.

side effects assessed

 SHP626 has been evaluated in preclinical and Phase 1 studies, in which the safety, tolerability and preliminary activity of SHP626 compared to placebo in healthy volunteers, as well as in overweight and obese volunteers, was assessed. The most common adverse events occurring in Phase 1 trials of SHP626 were gastrointestinal in nature, predominantly diarrhoea. While this occurred in most patients, it was not considered serious. There was one serious adverse event reported that was considered to be related to SHP626, alanine aminotransferase elevation, that led to discontinuation of the drug.

Additional information on the SHP626 Phase 2 study can be found on clinicaltrials.gov:

https://clinicaltrials.gov/ct2/show/NCT02787304?term=volixibat&rank=1