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The U.S. Food and Drug Administration has granted Orphan Drug Designation to Lymphoseek (technetium Tc 99m tilmanocept) Injection for use in sentinel lymph node detection in patients with head and neck cancers.

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Lymphoseek is a receptor-targeted, small-molecule radiopharmaceutical used for identifying lymph nodes that drain from a primary tumor that have the highest probability of being cancerous. Lymphoseek was approved earlier this year to guide sentinel lymph node testing for patients with head and neck cancers to help determine the extent of the spread of the tumor (OT 8/25/14 issue).


Lymphoseek's approval for head and neck cancer is based on a Phase III trial of 85 patients with squamous cell carcinoma of the lip, oral cavity, and skin, who were injected with Lymphoseek-the results showing that Lymphoseek-guided sentinel lymph node biopsy accurately determined if the cancer had spread through the lymphatic system.


Lymphoseek is also approved for use in patients with breast cancers or melanomas who are undergoing surgery to remove tumor-draining lymph nodes (OT 4/10/13 issue).


The manufacturer, Navidea Biopharmaceuticals, Inc., also received a Fast Track Small Business Innovation Research (SBIR) grant from the National Cancer Institute for up to $1.67 million to fund a trial to evaluate Lymphoseek in women with cervical cancer.


Also receiving the designation is RX-3117 for the treatment of patients with pancreatic cancer. RX-3117, marketed by Rexahn Pharmaceuticals, is a novel, small molecule anti-metabolite that is incorporated into DNA or RNA of cells and then inhibits DNA and RNA synthesis, killing tumor cells. Preclinical studies have shown that RX-3117 is active in cells that are resistant to gemcitabine, which is used to treat pancreatic cancer.


A Phase Ib clinical trial for the drug is currently enrolling patients and is expected to be completed in the first quarter of 2015.


The Orphan Drug designation-to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the U.S.-grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers.