Lumena Pharmaceuticals, a San Diego-based based company developing oral therapeutics for rare liver diseases, has received positive opinions for four Orphan Drug designations by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) for LUM001. The company's lead drug candidate, LUM001, received the first positive opinion granted by the EMA for primary sclerosing cholangitis (PSC). In addition, the company received positive opinions for LUM001 in three other rare cholestatic liver diseases, including Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and primary biliary cirrhosis (PBC). This follows the Orphan Drug Designation in September by the FDA Office of Orphan Product Development for LUM001 in the same indications.
"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," said Mike Grey, Lumena's chief executive officer and president. "We will continue to work diligently to develop LUM001 for the patients in serious need of safe, non-invasive therapeutic options to prevent progressive liver damage and the accompanying devastating symptoms."
Cholestatic liver diseases, such as 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. By reducing serum bile acids with a once-daily oral drug, LUM001 may offer a novel therapeutic approach to reducing elevated bile-acid levels, preventing liver damage and alleviating severe itching, which is generally the most debilitating symptom afflicting patients with these diseases.
LUM001 has been studied in more than 1,400 patients in 12 different clinical studies. Lumena currently is evaluating LUM001 in a phase II study in children with ALGS and a phase II study in adults with PBC. Later this year, Lumena plans to initiate a phase II study of LUM001 in children with PFIC, and a phase II study in adults with PSC.
The positive opinion of the COMP has now been forwarded to the E.U. commission for final approval. The EMA's Orphan Drug Exclusivity is designed to provide regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or debilitating conditions with a prevalence in the E.U. of not more than five in 10,000. Orphan medicinal products receive at least 10 years of market exclusivity in the E.U. after receiving marketing authorization.