FDA grants Elusys anthrax treatment orphan drug status

Orphan drug designation provides companies with financial and regulatory benefits during the course of the drug development including tax credits related to clinical trial expenses and a possible exemption from the FDA-user fee.

Anthim, which was granted fast track status in May 2005, is a high-affinity monoclonal antibody that targets the protective antigen component of anthrax, blocking the bacteria's ability to form deadly toxins.

The treatment is being developed under the FDA animal rule – a regulatory process specifically designed for the development of medical countermeasures to bioterror threats. According to this rule, marketing approval of Anthim can be granted based on efficacy in relevant animal models with an acceptable risk/benefit profile in humans.

In animal efficacy studies, Anthim demonstrated complete protection against an anthrax spore challenge with a single prophylactic dose and has shown significant protection when administered up to two days after a lethal challenge.

“Based on its impressive safety and efficacy data, Anthim is a valuable therapeutic for addition to the US Government's Strategic National Stockpile. Anthim's low therapeutic dose allows for rapid intramuscular (IM) delivery, the most effective mode of delivery to first-responders and civilian personnel in emergency situations,” said Dr Elizabeth Posillico, president and CEO of Elusys.

In 2005, the US biopharmaceutical company was awarded over $5 million from the National Institute of Allergy and Infectious Diseases and the Department of Defense for advanced formulation development. To date, the company has been awarded over $20 million from the US Government for the development of novel therapeutics to combat bioterror agents.