EC grants orphan drug status to Oxigene’s CA4P to treat neuroendocrine tumors

The designation gives the company 10 years of marketing exclusivity in European Union member countries if the product is approved.

Oxigene president and CEO William Schwieterman said: "This designation represents another successful step as we execute on our strategy of bolstering the proprietary position of CA4P in the potential indications in which we are most interested."

The final data from the company’s phase 2a clinical trial of CA4P in NETs is expected to be available later this year.

Earlier this year, CA4P was granted the orphan drug designation for NETs in the US.

CA4P (combretastatin-A4 phosphate or fosbretabulin) is Oxigene’s lead investigational vascular disrupting agent (VDA) product candidate. It is also being studied for the treatment of platinum resistant ovarian cancer.

Other tumor types including glioblastoma multiforme are being assessed for future studies.

CA4P targets established tumor blood vessels to exert its anti-tumor effects as a direct anti-vascular agent.

The company said CA4P selectively binds to the colchicine binding site of ß-tubulin which destabilizes the microtubules and results in sudden effects on the three-dimensional shape of the immature endothelial cells that are inadequately covered by pericytes.

When CA4P is used as a monotherapy, tumor regrowth is demonstrated on the peripheral rim of the tumor through recruitment of circulating endothelial progenitor cells. Oxigene said for this reason, it will focus on combination therapy with agents that inhibit the VEGF pathway.

Image: High magnification micrograph of a small intestine neuroendocrine tumour. Photo: courtesy of Nephron.