Lumena's rare liver disease candidate wins orphan status from FDA

09/30/2013 | Pharmaceutical Business Review Online

Lumena Pharmaceuticals' experimental drug LUM001 secured orphan-drug designation from the FDA as a treatment for Alagille syndrome, primary sclerosing cholangitis, progressive familial intrahepatic cholestasis and primary biliary cirrhosis. The drug is in a midstage pediatric trial for Alagille syndrome.

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