US Pharm. 2012;37(9)(Oncology/Hematology suppl):19.

Antisense Pharma GmbH announces that the FDA has granted orphan drug designation for its investigational oncology antisense compound trabedersen to treat malignant melanoma. Previously, trabedersen had received orphan drug designation by the European EMA and the FDA in high-grade glioma (malignant brain tumor) in 2002 and in advanced pancreatic cancer in 2009.

Philippe Calais, PhD, chief executive officer of Antisense Pharma, said, “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.”