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Candidate Anthrax Treatment Receives Orphan Drug Designation

By Michael Mair, March 10, 2006

On March 6, Medarex, Inc.(Medarex) and PharmAthene, Inc. (PharmAthene), announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation to Valortim™ (MDX- 1303).[1] Valortim™ is a fully human monoclonal antibody that Medarex and PharmAthene are co-developing to protect against inhalational anthrax.[2]

Valortim™ targets Protective Antigen -- one of the three components of the 'anthrax toxin.'[2] Neutralizing Protective Antigen inhibits the anthrax bacteria's virulence. In preclinical studies in animals, Valortim™ has been found to "provide significant protection against anthrax infection when administered prophylactically, or prior to the emergence of symptoms of anthrax infection, and also may increase survival when administered therapeutically, or once symptoms become evident."[1] In January 2006, FDA granted Fast Track designation to Valortim,™ and it is currently under evaluation in a Phase I clinical trial to evaluate the safety, tolerability, immunogenicity, and pharmacokinetics of a single dose administered intravenously or intramuscularly.[1,3]

A drug or biologic becomes and "orphan drug" when it receives orphan-drug designation from the FDA's Office of Orphan Products Development (OOPD), which administers the major provisions of the Orphan Drug Act (ODA).[4] The ODA provides incentives for sponsors to develop products for rare diseases or conditions. Under the ODA, a rare disease or condition is defined as any disease or condition that: (1) "affects less than 200,000 persons in the United States" or; (2) "affects more than 200,000 in the United States and for which there is no reasonable expectation that the cost of developing and making available in the United States a drug for such disease or condition will be recovered from sales in the United States of such drug."[5] An orphan drug designation qualifies the sponsor of the product to receive certain benefits from the federal government in exchange for developing the product, including tax credits, marketing incentives, and grants to help foster product research and development.[4,5]

References

1. PharmAthene and Medarex receive orphan drug designation for Valortim™ to treat anthrax infection. Medarex, Inc. March 6, 2006. Available at: http://www.medarex.com/cgi-local/item.pl/20060306-826999. Accessed March 10, 2006.
2. Valortim™ - prophylactic and therapeutic anti-anthrax monoclonal antibody (mab). Pharmathene, Inc. Available at: http://www.pharmathene.com/valortim.html. Accessed March 10, 2006.
3. PharmAthene and Medarex receive FDA fast track designation for Valortim™ for treatment of anthrax infection. Medarex, Inc. January 9, 2006. Available at: http://www.medarex.com/cgi-local/item.pl/20060109-801756. Accessed March 10, 2006.
4. Orphan product designation. U.S. Food and Drug Administration, Office of Orphan Products Development. Available at: http://www.fda.gov/orphan/designat/index.htm. Accessed March 10, 2006.
5. The Orphan Drug Act (as amended). U.S. Food and Drug Administration, Office of Orphan Products Development. Available at: http://www.fda.gov/orphan/oda.htm. Accessed March 10, 2006.