Purdue Pharma announced that Imbrium Therapeutics LP, its’ clinical stage biopharmaceutical subsidiary, and the Swiss company Mundipharma EDO GmbH have received orphan drug designation from the U.S. Food and Drug Administration for their investigational drug etoposide toniribate, a novel topoisomerase II inhibitor designed for the treatment of relapsed refractory biliary tract cancer.
Orphan drug designation is given to drugs and biological products intended for the treatment of rare diseases or disorders that impact fewer than 200,000 people in the U.S. Biliary tract cancer, also known as cholangiocarcinoma, is a rare cancer that starts in the bile ducts and mostly impacts patients 65 and older. Roughly 8,000 people in the U.S. are diagnosed with the disease each year.
“We are pleased that the FDA has granted orphan drug designation for etoposide toniribate as we believe, once approved, it can be an important clinical advance for patients suffering from relapsed refractory biliary tract cancer, a patient population that has limited treatment options,” said Paul Medeiros, president of Imbrium Therapeutics. “This designation represents Imbrium’s first milestone in oncology and underscores our commitment to advance the clinical development of oncology chemotherapeutics while actively collaborating to advance treatments across our therapeutic portfolio.”
The European Medicines Agency granted orphan designation to etoposide toniribate on June 4, 2014. Imbrium Therapeutics and Mundipharma EDO GmbH plan to begin a Phase 3 clinical trial of etoposide toniribate in patients with refractory biliary tract cancer in 2020.