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Battelle Life Sciences Research CRO+ Today

Battelle Life Sciences Research CRO+ Today

April 2016 - Issue 6

News
Bacillus anthracis and Bacillus subtilis Spores

FDA Approves New Anthrax Antitoxin Tested at Battelle

A powerful new treatment option for inhalational anthrax is now available, thanks in part to researchers at Battelle. ANTHIM (obiltoxaximab) Injection, patented and produced by Elusys Therapeutics, was licensed by the FDA in March. Battelle conducted the required efficacy research under the FDA Animal Rule to help bring it to market.

ANTHIM will be an important addition to the Strategic National Stockpile (SNS), which stores large quantities of medicines, therapies and vaccines to protect the public in the case of an epidemic, natural disaster or terrorist attack. It will be given in combination with antibiotics to victims who have been exposed to anthrax to counter the toxins produced by the bacteria. 

Anthrax, caused by the Bacillus anthracis bacterium, is highly lethal but rarely seen naturally in developed countries. However, its combination of lethality and hardiness make it an attractive option as a bioweapon. Several foreign governments are known to have developed weaponized versions. This makes development of antitoxins of critical importance to protect warfighters and the public in the event of a terrorist attack.

Because the disease is rare and lethal, it is not feasible or ethical to conduct clinical trials for efficacy in humans. ANTHIM was approved under the FDA Animal Rule, which provides an alternative licensure pathway for products that cannot be tested in humans.

Elusys came to Battelle for help in conducting efficacy trials for the antitoxin using alternative animal models. Researchers at the Battelle Biomedical Research Center conducted various studies using two animal models, led by Principal Investigator and Study Director Dan Sanford and Study Director Lisa Henning. The work was awarded under a contract with Elusys that was funded by the Biomedical Advanced Research and Development Authority (BARDA), National Institutes of Health (NIH) and Department of Defense (DoD).

Battelle has extensive experience with the FDA Animal Rule. Only four products have been awarded “in-class” approval under the rule, meaning they were undergoing approval for the first time and for their primary intended purpose. (Other products approved through the Animal Rule were already on the market and had previously completed human clinical trials for other uses.) Battelle played a leading role in the approval of all four products, including Raxibacumab, another anthrax antitoxin and the very first product to achieve in-class approval under the alternative pathway. Battelle researchers also conducted non-clinical research for AIGIV (now marketed as Anthrasil), a third anthrax antitoxin, and BAT, a botulism antitoxin.

Conducting efficacy studies as replacements for human clinical trials is highly complex. Researchers must identify an alternative animal model for the disease that demonstrates similar disease progression, and drug pharmacokinetics should be similar to those observed in healthy human subjects. Once the therapeutic dose has been established for the alternative models, sophisticated statistical and pharmacokinetic methods are used to select an appropriate effective dose for humans. 

Battelle has a long history in animal model development and validation for a wide variety of pathogens. It is also home to the largest independent biocontainment facility in the world (BLS-3), with capabilities to support highly regulated GLP studies that fully meet FDA requirements. They bring together a unique range of life science research capabilities, including assay development and validation, aerosol science, microbiology, molecular immunology, clinical pathology, anatomical pathology and toxicology. Because of the depth and breadth of experience on their research teams, Battelle is often trusted with complex studies that are beyond the capabilities of standard contract research organizations.   

Battelle will be able to apply the methods developed and lessons learned for this study to other research under the FDA Animal Rule. Trials are already underway for new vaccines and therapies against other deadly pathogens, including Yersinia pestis (plague), smallpox, botulism and tularemia.