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Lumena Pharmaceuticals Receives Orphan Drug Designation from US Food & Drug Administration for LUM001 in Four Rare Liver Diseases
Date:9/26/2013

nt 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." 

LUM001 has been studied in more than 1,400 patients in 12 different clinical studies. The company also announced today in a separate announcement the initiation of a Phase II study of LUM001 in children with ALGS. The company will also soon be initiating a Phase II study in adult patients with PBC. Later this year, Lumena plans to initiate a Phase II study of LUM001 in pediatric patients with PFIC, and a Phase II study in adults with PSC.

Under the Orphan Drug Act, the U.S. FDA may grant Orphan Drug Designation to a drug intended to treat rare diseases, generally those which affect fewer than 200,000 individuals in the country. Lumena is also pursuing Orphan Drug Designation for LUM001 in all four indications with the European Medicines Agency.

About Alagille Syndrome

ALGS is a rare genetic disorder that can affect the liver, heart, and other parts of the body. Approximately one in every 70,000 children is born with ALGS. In patients with ALGS the bile ducts are abnormally narrow, malformed and reduced in number. This prevents normal bile flow from the liver into the small intestine. As a result, bile builds up in the liver, causing damage that prevents the liver from working properly. ALGS is characterized by elevated bile acids and severe itching, which is generally the most debilitating symptom afflicting children with this liver disease. Treatment with anti-pruritics (anti-itching drugs) typically provides only modest relief. Procedures that remove bile from the circulation can lower serum bile acids, reducing itch and improving liver function in some patients, but tend to be invasive in nature.

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