Drug discovery company Cleveland BioLabs (NASDAQ: CBLI) said Tuesday that CBLB502, a drug under development to treat exposure to radiation, has been granted orphan drug status by the US FDA for the prevention of death following a potentially lethal dose of radiation after a nuclear disaster.
The orphan drug designation is given to drugs that are intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US, or that affect more than 200,000 but are not expected to recover the costs of developing or marketing the treatment.
The status qualifies CBLB502 for an accelerated review process, tax credits, financial assistance for development costs, and seven years of marketing exclusivity. The designation also allows for a possible exemption from the FDA-user fee and assistance in clinical trial protocol design.
"We are very excited that CBLB502 has been assigned Orphan Drug status for its lead indication and look forward to working closely with the FDA as we complete development," said president and CEO Michael Fonstein, Ph.D..
In July, CBLB502 was granted fast track status from the FDA for reducing the risk of death following total body irradiation during or after a radiation disaster, as there is currently no FDA-approved medical countermeasure for this.
CBLB502 is a bio-engineered derivative of a microbial protein that potentially reduces injury from acute stresses, such as radiation and chemotherapy, by mobilizing several natural cell protective mechanisms, including the inhibition of cell death, the reduction of oxidative damage, and the induction of regeneration-promoting cytokines.
The company said that results from a phase 1 human study indicated that the drug was generally well tolerated and that results corresponded to those previously seen in animals, with a rapid and potent cytokine response.