Orphan drug designation is available in Europe as an incentive to provide regulatory and financial assistance to companies to research medical treatments and drugs intended to treat life-threatening or chronically debilitating conditions.
CTI president Craig W Philips said that with the pixantrone new drug application review progressing well in the US, the company is moving forward with the marketing approval application process in Europe.
“Orphan drug designation in Europe, if granted, could provide important regulatory, financial and commercial advantages for CTI,” Philips said.
Pixantrone is a novel topoisomerase II inhibitor, which utilises a different molecular structure that differentiates it from anthracyclines and other related chemotherapy agents.
As a result the treatment does not suffer the same limitations as anthracyclines, which can cause cumulative heart damage and are largely ineffective for people whose cancer has returned after previous anthracycline treatment.
CTI initiated the process of obtaining marketing approval in Europe in July 2009 and is expecting a formal Marketing Authorization Application (MAA) in 2010, following approval of the paediatric investigation plan which was submitted to the EMEA earlier in September.
CTI would be granted ten-year market exclusivity in Europe if the MAA is approved.
Pixantrone is under review for approval by the US Food and Drug Administration.