Cancer Immunotherapy Receives Orphan Drug Designation from FDA

The US FDA notified the Austrian biotech company Activartis on 26^th June that Orphan Drug Designation (ODD) has been granted for its innovative Cancer Immunotherapy AV0113. The ODD applies specifically to the use of AV0113 for the treatment of malignant glioma, a very aggressive type of brain cancer. The European drug agency EMA already granted ODD at the end of last year.

In principle, Activartis’ AV0113 Cancer Immunotherapy may be used to fight any type of cancer. The individualised therapeutic technology is based on a patented procedure in which a cancer patient’s immune system is primed to fight the tumor and eventually control its growth. This concept is based on the use of Dendritic Cells, the key regulatory elements of the immune system, that are the same as the tumor tissue derived from the patient.

AV0113 activates the patient’s immune system, with tumour cells being identified on the basis of their antigens and destroyed. The therapy makes use of elements and mechanisms of the immune system and gets to work where these fail. As tumour cells are the body’s own tissue, the immune system does not normally identify them as dangerous. Activartis’ AV0113 Cancer Immunotherapy ‘tricks’ Dendritic Cells and, consequently, a cancer patient’s immune system, into doing the right thing, i.e. to perceive the tumour as a threat and to trigger adequate defense mechanisms.

At the beginning of 2013, Activartis completed recruitment of 78 brain cancer patients to a multi-centre, randomised, phase II clinical trial. This randomised study aims to deliver safety and efficacy data for the first time. Preliminary results presented at the AACR Annual Meeting revealed a very promising trend suggesting a survival benefit of patients in the AV0113 treatment group compared to the randomised control group.

Confirmation of that trend is expected by the end of 2013. If the trend currently observed is confirmed, AV0113 is bound to become part of the standard therapy for GBM.