The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Avacta Group’s chemotherapy drug, AVA6000, for the treatment of soft tissue sarcoma.
AVA6000 is a form of the generic chemotherapy doxorubicin that has been modified using pre|CISION technology so that it is activated predominantly in the tumour with the aim of sparing healthy tissue from exposure and improving the safety, tolerability and efficacy of the drug.
Avacta has an ongoing Phase I clinical trial to assess the safety and pharmacokinetics of AVA6000, which has potential as a treatment for patients with a range of cancer types. The trial recently advanced to the fourth dose cohort of patients at 200mg/m2, following a positive review of the safety and tolerability data from the third cohort.
The FDA can grant ODD based on a review of preclinical data from investigational treatments for rare diseases, which are defined as conditions affecting fewer than 200,000 people in the US. This designation qualifies the developer of the drug for certain incentives, like seven years of market exclusivity upon drug approval from the FDA.
Soft-tissue sarcoma is a rare mesenchymal malignancy which accounts for less than 1% of all adult tumours. Despite the successful advancement of localised therapies, such as surgery and radiotherapy, these tumours can recur, often with metastatic disease.