Antisense Pharma GmbH has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation for its investigational oncology antisense compound trabedersen to treat malignant melanoma.
Previously, trabedersen has received Orphan Drug designation by the European EMA and the US FDA in high-grade glioma (malignant brain tumor) in 2002 and in advanced pancreatic cancer in 2009.
Dr. Philippe Calais, Chief Executive Officer of Antisense Pharma comments: "We are delighted that with this most recent designation by the FDA, Trabedersen has now orphan status for three different cancer indications. With the orphan status and the associated marketing protection, we have achieved a core milestone for the long-term commercial success of our oncology lead compound trabedersen."
Dr. Hubert Heinrichs, Chief Medical Officer of Antisense Pharma elaborates: "Malignant melanoma is a very aggressive disease with a high need for innovative, effective and well-tolerated treatment options. We are confident that traberdersen will provide new hope to patients that suffer from malignant melanoma. Recently published results of our Phase I/II Study1 with traberdersen demonstrated excellent tolerability and initial encouraging survival data in patients with advanced tumors. We are currently evaluating further clinical development of traberdersen in malignant melanoma, preferably together with a potential partner."