SuperGen Inc announced that the company's Partaject- delivered busulfan has received orphan-drug designation from the Food and Drug Administration (FDA) for the preparative therapy of pediatric patients undergoing bone marrow transplantation. As such, SuperGen will be entitled to seven years of market exclusivity from the time Partaject-delivered busulfan receives FDA marketing approval for this indication.

SuperGen's Partaject delivery system allows compounds that are insoluble or poorly soluble in water to be delivered intravenously (or by other routes) without the need for organic solvents that may in themselves be toxic. Busulfan is used in combination therapy prior to allogeneic bone marrow transplants (from a donor, rather than from oneself) in patients with chronic myelogenous leukemia.

"We are pleased to have received orphan-drug designation for Partaject-delivered busulfan as a preparative treatment for pediatric bone marrow transplants," said Dr. Joseph Rubinfeld, chairman and chief executive officer of SuperGen. "While bone marrow transplants have saved thousands of lives, many patients have suffered complications, some of which can be fatal. We believe that Partaject-delivered busulfan may offer new hope to the young patients undergoing this procedure.

"Today's announcement is another step in establishing SuperGen as the premier oncology company," added Dr. Rubinfeld. "We are delighted that Partaject-delivered busulfan is part of our broad and expanding pipeline of products designed to treat a variety of solid tumors and haematological malignancies."

Earlier this year, data from a Phase I clinical study of Partaject-delivered busulfan suggested the drug is safe and effective in patients with neoplastic meningitis, an end-stage condition where 5 to 30 per cent of all solid tumors metastasize to the meninges area of the brain.