CuraGen Corporation, a genomics-based pharmaceutical company, announced that the US Food and Drug Administration (FDA) has granted orphan drug designation to CG53135 for the treatment of radiation induced oral mucositis (OM).

This investigational protein, a novel growth factor discovered by CuraGen's integrated platform, promotes epithelial and mesenchymal cell proliferation in vitro and is active in animal models of OM. CG53135, CuraGen's lead product candidate, is currently in Phase I clinical trials to evaluate the safety and pharmacokinetics in patients with cancer who are at risk for mucositis. CuraGen expects to initiate Phase II trials with CG53135 in patients with OM in 2004.

"We believe the strength of CG53135 resides in its ability to promote both epithelial and mesenchymal cell proliferation possibly resulting in increased activity," stated Timothy Shannon, chief medical officer of CuraGen. "The FDA's orphan drug designation further strengthens our programme for CG53135 by offering clinical development and commercialization benefits."

The Orphan Drug Act is intended to encourage companies to develop therapies for the treatment of diseases that affect fewer than 200,000 individuals in the US at the time of application. Further criteria include the ability of the product to address an unmet medical need where no approved treatment option exists or provide significant benefit over available treatments. Orphan drug designation, granted by the FDA's Office of Orphan Products Development, provides CuraGen with a number of potential benefits for CG53135. Orphan drug designation may result in seven years of market exclusivity in the United States upon FDA product approval, provided that the sponsor company continues to meet certain conditions established by the FDA. Upon marketing authorization and during the period of market exclusivity, the FDA does not accept or approve other applications to market the same medicinal product for the same therapeutic indication. Other incentives provided by orphan designation include protocol assistance and eligibility for research and development support. Protocol assistance includes regulatory assistance and reduced filing fees, as well as advice on the conduct of clinical trials.

Oral mucositis is a side effect experienced by cancer patients undergoing chemotherapy or radiation therapy. The disease is characterized by inflammation and ulceration of the tissue lining the mouth and throat, leading to bleeding, pain, and difficulty eating and drinking. Oral mucositis is sometimes a cause for clinicians to interrupt patients' cancer treatment regimens, thus limiting the success of therapeutic plans. An effective treatment for oral mucositis has the potential to treat debilitating symptoms, to allow cancer patients to tolerate the appropriate doses of cancer treatment, and to decrease hospitalization time. There is currently no FDA approved therapy to treat this condition.