Antisense Receives Orphan Drug Designation From FDA, EMEA For Trabedersen

Antisense Pharma (Antisense) has received orphan drug designation from both the European Medicines Agency (EMEA) and the FDA for its investigational drug, trabedersen, in the treatment of pancreatic carcinoma.

Trabedersen has already been granted orphan drug designation by both the authorities in the treatment of high-grade gliomas in 2002.

In an ongoing clinical phase I/II study, trabedersen has shown a good safety and tolerability profile and encouraging survival data in patients with advanced pancreatic carcinoma.

The Committee on Orphan Medicinal Products (COMP) of the EMEA in its report said: “The preliminary clinical data are quite impressive.”

Reportedly, 23 patients received single agent trabedersen intravenously as second-, third-, or fourth-line treatment, either in a 7-day on/7-day off or 4-day on/10-day off schedule.

In the same study, good safety, tolerability and encouraging first efficacy data for trabedersen was observed in patients with advanced malignant melanoma or colorectal carcinoma.

Karl-Hermann Schlingensiepen, CEO of Antisense, said: “We are delighted to have received the orphan drug status from the EMEA and FDA for the treatment of pancreatic carcinoma. This further accelerates our efforts to make trabdersen available to those who need it as quickly as possible.”

“Based on the results of several clinical studies, we expect trabedersen to significantly improve the therapeutic outcome not only of patients with pancreatic carcinoma but also of patients suffering from high-grade gliomas, malignant melanoma or colorectal carcinoma. Indeed, with its unique mode of action, we believe that trabedersen has the potential to lead to a paradigm shift towards tackling malignant tumors at their roots while providing a better quality of life for patients.”