Guerbet has announced that it has been granted Orphan Drug Designation from the US Food and Drug Administration Office of Orphan Products Development (OOPD) for Lipiodol (ethiodised oil) injection for management of patients with known hepatocellular carcinoma.
Hepatocellular carcinoma prevalence in the USA is estimated to affect approximately 35,000 patients in 2013. Orphan Drug Designation is granted to drug therapies intended to treat diseases or conditions that affect fewer than 200,000 people in the United States. Orphan Drug Designation entitles the sponsor to clinical protocol assistance with the FDA, as well as federal grants, tax credits, and potentially a seven year market exclusivity period.
“We are very pleased to have been granted an orphan drug designation for Lipiodol,” commented Yves L’Epine, chief executive officer of Guerbet. “Patients with known hepatocellular carcinoma may have Lipiodol approved as part of their disease management as an option.”
Lipiodol is currently under FDA evaluation for safety and efficacy with a proposed indication “for selective hepatic intraarterial use in computed tomography of the liver to visualise and localise lesions in adults with known hepatocellular carcinoma.”