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LUM001 Receives Orphan Drug Designation for Treatment of Rare Liver Diseases
The FDA has granted an Orphan Drug Designation for the investigational compound LUM001 for the treatment of four rare cholestatic liver diseases, including Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), primary biliary cirrhosis (PBC), and primary sclerosing cholangitis (PSC).
Cholestatic liver diseases, such as ALGS, PFIC, PBC, and PSC, result in impaired bile acid flow and the retention of these acids in the liver, leading to progressive liver damage that can cause liver failure. According to the drug’s developer (Lumena Pharmaceuticals), once-daily treatment with LUM001 may offer a new therapeutic approach to reducing elevated bile acid levels, thereby preventing liver damage and alleviating severe itching, which is generally the most debilitating symptom affecting patients with these diseases.
Under the Orphan Drug Act, the FDA may grant an Orphan Drug Designation to a drug intended to treat rare diseases, generally those which affect fewer than 200,000 individuals in the country. LUM001 is an inhibitor of the apical sodium-dependent bile acid transporter (ASBT), which recycles intestinal bile acids back into the circulation. The drug has been studied in 12 clinical trials involving more than 1,400 subjects.[Source: Lumena Pharmaceuticals; September 26, 2013]