Irish biopharmaceutical company Shire has been granted the US Food and Drug Administration (FDA) Fast Track designation for SHP626 (volixibat) to treat non-alcoholic steatohepatitis (NASH) with liver fibrosis.
SHP626 (volixibat) can be used for the investigational treatment of NASH-affected adults with liver fibrosis.
NASH is a type of serious, chronic and non-alcoholic fatty liver disease (NAFLD), for which no drugs have currently been approved.
The disease causes inflammation and accumulation of fat in the liver, thereby leading to diseases such as fibrosis, liver failure, cirrhosis and liver cancer.
SHP626 is being developed as a once-daily, orally administered inhibitor of the apical sodium dependent bile acid transporter (ASBT), a protein mainly responsible for recycling bile acids from the intestine to the liver.
Shire research and development (R&D) head Dr Philip J. Vickers said: “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.
“This fast track designation is further recognition of the critical need to develop new, effective therapeutic options for patients with this serious condition.”
The US FDA Fast Track Designation for SHP626 in NASH was supported by preclinical and Phase I studies, which revealed that the drug showed symptoms of one serious adverse effect alanine aminotransferase elevation, resulting in its discontinuation.
Shire will commence its Phase II trial with SHP626 as a randomised, placebo-controlled, double-blind study to assess the safety, tolerability and efficacy of three doses of volixibat over 48 weeks in adult patients with NASH.
The Phase II study will be conducted in the US, Canada and the UK.