Lumena announced that the FDA has granted Orphan Drug designation to LUM001 in four rare cholestatic liver diseases: Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), primary biliary cirrhosis (PBC), and primary sclerosing cholangitis (PSC). LUM001 is an inhibitor of the apical sodium-dependent bile acid transporter (ASBT), which recycles intestinal bile acids back into the circulation.
LUM001 has been studied in more than 1,400 patients in 12 different clinical studies. Lumena plans on conducting four separate Phase 2 studies of LUM001 in children with ALGS, in adult patients with PBC, in pediatric patients with PFIC, and in adults with PSC.
For more information call (858) 461-0694 or visit LumenaPharma.com.