FDA Grants Orphan Drug Status To Emergent's BioThrax

Emergent BioSolutions announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to its drug BioThrax (Anthrax Vaccine Adsorbed) for post-exposure prophylaxis (PEP) of anthrax disease caused by exposure to Bacillus anthracis.

BioThrax is the only FDA-licensed vaccine for the prophylaxis of anthrax disease. The drug is currently licensed for the indication of pre-exposure prophylaxis of anthrax. The company has recently completed a non-interference study funded by the Biomedical Advanced Research and Development Authority (BARDA) within the Office of the Assistant Secretary for Preparedness and Response in the U.S. Department of Health and Human Services. Results from the study will be used to support a supplemental BLA seeking licensure of a PEP indication for BioThrax, for use in combination with antibiotics in people with suspected or confirmed exposure to anthrax.

Anthrax is a potentially deadly disease resulting from exposure to spore-forming bacterium Bacillus anthracis. Anthrax spores germinate into bacteria once inside the body and secrete three proteins. These are protective antigen, lethal factor, and edema factor. The proteins are non-toxic individually but can turn lethal when combined and interact on and within the cells of the exposed organism.

Adam Havey, EVP and President of biodefense division at Emergent BioSolutions, said, “Emergent is pleased with FDA’s Orphan Drug Designation of BioThrax for post-exposure prophylaxis. This designation will help streamline discussions around regulatory requirements at our pre-BLA meeting with FDA next month. We look forward to discussing our supplemental application for the expanded indication of post-exposure prophylaxis and the role of BioThrax in the treatment of inhalation anthrax.”

Orphan drug designation is granted to drugs and biologics being developed specifically for rare medical conditions, in particular those that affect fewer than 200,000 people in the U.S. The designation provides a range of incentives to Emergent’s BioThrax PEP Program, including the waiver of the Biologics License Application (BLA) supplemental regulatory filing fee and up to seven years of marketing exclusivity if approved.