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FDA Actions & Updates

The latest approvals, designations, and new indications from the U.S. Food and Drug Administration for oncology drugs.

Monday, February 25, 2019

Orphan Drug Designation Granted for Etoposide Toniribate in Relapsed/Refractory Biliary Tract Cancer

The FDA has granted Orphan Drug Designation (ODD) to etoposide toniribate for the treatment of relapsed/refractory biliary tract cancer, also known as cholangiocarcinoma.

Biliary tract cancer is a rare tumour with approximately 8,000 patients diagnosed in the U.S. every year and 10,571 in Europe. The FDA grants ODD status to medicines intended for the treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Radical surgery is the only curative treatment for biliary tract cancer but, in most cases, the cancer is inoperable. Patients who fail first-line chemotherapy have limited treatment options and the standard of care is generally palliative. The 5-year survival rates for patients with biliary tract cancer are very low, approximately 15 percent.

Etoposide toniribate has shown encouraging data in phase II trials and this data has been key in securing the ODD. This news is in addition to the European Medicines Agency (EMA) also granting orphan designation on June 4, 2014. As with the FDA, this designation allows protocol assistance, reduced fees and 10-years marketing exclusivity in that disease after approval. It can also lead to speedier reimbursement in many EU member states.

Etoposide toniribate is to be accelerated through a phase III trial, with the aim of providing a potential additional treatment option in this area of unmet patient need.