Alfacell Files for Orphan Drug Designation for ONCONASE®

January 3, 2007 - New Jersey Business News reports that Alfacell Corporation has filed an application with the U.S. Food and Drug Administration (FDA) to obtain orphan designation for its lead drug candidate ONCONASE® (ranpirnase) for the treatment of patients with unresectable malignant mesothelioma (UMM). ONCONASE is currently being evaluated in a confirmatory Phase IIIb clinical trial in UMM.

The orphan drug designation provides incentives to pharmaceutical and biotechnology companies to develop drugs for the treatment of diseases affecting fewer than 200,000 people in the United States. There are about 4,000 new cases on unresectable malignant mesothelioma reported in the U.S. each year, therefore qualifying it as an orphan disease.

Kuslima Shogen, Chief Executive Officer of Alfacell, said, "Orphan drug designation is an important tool for companies like Alfacell that are developing drugs for rare and emerging diseases. This is why we pursued and were granted orphan designation for ONCONASE for the mesothelioma indication in Europe and Australia. As such, we believe we are well-positioned with FDA for review of our application.”

“With the U.S. orphan drug application filed and over 385 patients enrolled in the pivotal UMM study, we are continuing to make progress on the critical path leading to the anticipated completion of the NDA submission for ONCONASE, pending positive data, in mid-2007,” Shogen added.

Orphan drug status will allow Alfacell seven years of marketing for ONCONASE exclusively for the UMM indication, once the FDA has approved the drug. The newspaper report also notes that Alfacell would receive “protocol assistance by the FDA in the preparation of a dossier that will meet regulatory requirements, tax credits, grant funding for research and development, and reduced filing fees for the marketing application.”