The US Food & Drug Administration (cof Orphan Product Development has granted orphan drug designation for Lumena Pharmaceuticals' LUM001 clinical program.
Lumena obtained orphan designation for LUM001 in four rare cholestatic liver diseases which include Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), primary biliary cirrhosis (PBC), and primary sclerosing cholangitis (PSC).
The company has studied LUM001in over 1,400 patients in 12 different clinical studies.
Lumena said cholestatic liver diseases like ALGS, PFIC, PBC and PSC, result in impaired bile acid flow and retention of bile acids in the liver, leading to progressive liver damage that can cause liver failure.
According to the company, LUM001 may provide a novel therapeutic approach to reducing elevated bile-acid levels, preventing liver damage and alleviating severe itching by decreasing serum bile acids with a once-daily oral drug.
Lumena Pharmaceuticals chief medical officer Alejandro Dorenbaum said orphan drug designation will enable the company to pursue an accelerated pathway for development of LUM001.
"Current therapeutic options for cholestatic liver diseases are of limited efficacy, and surgical approaches to reducing bile acid levels to manage disease symptoms can be disfiguring and prone to complications. The only remaining option for many patients is liver transplant," Dorenbaum added.
The company has already started a Phase II study of LUM001 in children with ALGS.
Lumena is also planning to start a Phase II study in adult patients with PBC shortly and later in 2013, the company expects to initiate a Phase II study of LUM001 in pediatric patients with PFIC, and a Phase II study in adults with PSC.