Advaxis, Inc., a clinical-stage biotechnology company developing cancer immunotherapies, announced that the European Medicines Agency (EMA) granted Orphan Drug designation for ADXS-HER2 for the treatment of osteosarcoma.

“Receiving Orphan Drug designation from the EMA is another significant step forward for Advaxis as we continue to advance ADXS-HER2,” said Daniel J. O'Connor, CEO of Advaxis.

“We recently initiated our first-in-human study of our lead Lm Technology immunotherapy product for HER2 expressing solid tumors and we hope to expand that trial into four HER2 expressing tumor types, including breast, gastric, esophageal and osteosarcoma.”

Orphan Drug designation in the EU is granted to drugs or biologics that treat a life-threatening or chronically debilitating rare disease affecting fewer than five in 10,000 individuals in the European Union. Products receiving orphan drug designation are eligible to receive market exclusivity for a period of up to ten years, as well as development incentives such as regulatory and protocol assistance and scientific advice.

Human epidermal growth factor receptor 2 (HER2) is overexpressed in a percentage of solid tumors such as breast, gastric, bladder, brain, pancreatic, ovarian and paediatric bone cancer (osteosarcoma). The American Cancer Society estimates that in 2015 in the United States alone there will be 231,840 new cases of invasive breast cancer; 24,590 new cases of gastric cancer; 74,000 new cases of bladder cancer; 22,850 new cases of brain/spinal cancer; 48,960 new cases of pancreatic cancer; 21,290 new cases of ovarian cancer; and 207 new cases of paediatric osteosarcoma. HER2 expression is associated with more aggressive disease, increased risk of relapse and decreased overall survival, and is an important target for immunotherapy.

ADXS-HER2 is an Lm Technology immunotherapy product candidate being developed by Advaxis to target HER2 expressing cancers. ADXS-HER2 has received orphan drug designation by the US Food and Drug Administration (FDA) for the treatment of osteosarcoma. Advaxis is developing ADXS-HER2 for both human and animal health, and has seen encouraging data in canine osteosarcoma, which is considered a model for human osteosarcoma. Advaxis has licensed ADXS-HER2 and three other immunotherapy constructs to Aratana Therapeutics, Inc. for the development of pet therapeutics.