EMEA recommends orphan drug designation for Progen drug

The agency submitted its opinion to the European Commission (EC) for orphan designation determination. This will become effective upon adoption of the recommendation by the EC, which is expected to occur before the end of the third quarter of 2007.

The EMEA’s orphan drug program is designed to promote the development of drugs to treat rare life-threatening or very serious conditions that affect no more than five in every 10,000 people in the European Union (EU). The designation provides EU market exclusivity for up to ten years in the given indication.

PI-88 is part of a new class of multi-targeted cancer therapeutics inhibiting both angiogenesis (or tumour promoting) factors such as vascular endothelial growth factor (FEGF), fibroblast growth factors (FGF) 1 and 2, and heparanase, a degrading enzyme implicated in metastasis (tumour spread).

Progen will this year launch a multi-national two-armed, double-blinded placebo controlled Phase III trial using PI-88, with the primary endpoint of disease free survival, in patients with post-operative primary liver cancer. The goals of PI88 treatment in this population are to reduce disease recurrence, prolong the time to recurrence (known as disease-free survival time) and improve the overall survival time of patients after tumour resection.

Additionally, the company is currently conducting a Phase II trial in patients with melanoma, the other indication in which it has received US FDA orphan drug designation, with results from this trial expected during the second half of 2008.

Mr Justus Homburg, CEO of Progen commented: “While the incidence of primary liver cancer in the EU is relatively low, with over half a million new cases elsewhere in the world, it is a disease of significant global relevance.”