Friday, July 29, 2016

Shire’s SHP626 (Volixibat) Receives FDA Fast Track for Adults who have NASH with Liver Fibrosis

Shire’s SHP626 (Volixibat) Receives FDA Fast Track Designation for an Investigational Treatment for Adults who have Nonalcoholic Steatohepatitis (NASH) with Liver Fibrosis

July 29, 2016

Lexington, Mass. – July 29, 2016 – Shire plc (LSE: SHP, NASDAQ: SHPG) today announced that the United States Food and Drug Administration (FDA) has granted Fast Track designation for SHP626 (volixibat) for an investigational treatment of adults who have nonalcoholic steatohepatitis (NASH) with liver fibrosis. Shire is developing SHP626 as a once daily, orally-administered inhibitor of the apical sodium dependent bile acid transporter (ASBT), a protein which is primarily responsible for recycling bile acids from the intestine to the liver. NASH is a serious, chronic liver disease for which 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. "This Fast Track designation is further recognition of the critical need to develop new, effective therapeutic options for patients with this serious condition." 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 randomized, placebo-controlled, double-blind study to evaluate the safety, tolerability and efficacy of three doses of volixibat over 48-weeks in adult patients with NASH. The Phase 2 study will be conducted in the U.S., Canada and the United Kingdom.

Additional information on the SHP626 Phase 2 study can be found on

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 diarrhea. While this occurred in most patients, it was not considered serious. There was one serious adverse event reported that was considered related to SHP626, alanine aminotransferase elevation, that led to discontinuation of drug.

About Nonalcoholic Steatohepatitis (NASH)

NASH is a type of nonalcoholic fatty liver disease (NAFLD), characterized by inflammation and the accumulation of fat in the liver, for which there are currently no approved drugs. It can be severe and lead to fibrosis, cirrhosis, liver failure and liver cancer. There is a steady rise in the prevalence of NASH in the U.S. and globally, and the disease is typically associated with obesity, type 2 diabetes, hypertension, high cholesterol and triglycerides. NASH is currently the second leading cause of liver transplantation in adults in the U.S., and is estimated to become the leading cause for liver transplantation if the current trajectory continues.

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