Potential treatment for rare liver diseases moves forward in EU

The European Medicines Agency Committee for Orphan Medicinal Products issued a positive opinion for orphan drug designation on Lumena Pharmaceuticals’ lead drug candidate, according to a company news release.

The candidate, LUM001, received a positive opinion for treatment of Alagille syndrome, progressive familial intrahepatic cholestasis, primary biliary cirrhosis and primary sclerosing cholangitis. It has been forwarded to the EU commission for final approval.

LUM001, a once-daily oral drug designed to reduce serum bile acid, has been analyzed in 12 clinical studies involving more than 1,400 patients. It currently is undergoing a phase 2 trial in children with Alagille syndrome and a phase 2 study in adults with primary biliary cirrhosis. Two additional phase 2 trials — a study in children with progressive familial intrahepatic cholestasis and another in adults with primary sclerosing cholangitis — are scheduled to begin shortly.

“The EMA’s recognition of LUM001 as a potential treatment for the thousands of patients who suffer from these debilitating, rare liver diseases is another important step in our development plans,” Mike Grey, Lumena’s chief executive officer and president, said in the release.

The FDA granted orphan drug designation to LUM001 — an inhibitor of the apical sodium-dependent bile acid transporter, which recycles intestinal bile acids back into the circulation — for the same indications in September.

Orphan designations are designed to create regulatory and financial incentives for companies to develop potential treatments for serious, but rare conditions that affect fewer patients.