Trabedersen granted orphan drug designation from both FDA And EMEA for treating pancreatic carcinoma

Sep 14 2009

The biopharmaceutical company Antisense Pharma GmbH has announced today that it has received orphan drug designation from both the European Medicines Agency EMEA and the US Food and Drug Administration FDA for its investigational drug trabedersen in the treatment of pancreatic carcinoma. Trabedersen has already been granted orphan drug designation by both authorities in the treatment of high-grade gliomas in 2002. This underlines the high potential of trabedersen to treat various aggressive tumors.

Orphan drug designation can be applied for if the disease is life-threatening or chronically debilitating and affects not more than five in 10,000 persons in the European Community (equals around 250,000 people) and fewer than 200,000 people in the United States, respectively, in case that no other satisfactory therapy exists or the medicinal product is expected to provide significant benefit over existing therapies.

The orphan drug designation is meant to encourage pharmaceutical companies to develop drugs for diseases that meet the above criteria by providing them scientific advice, reduction or waiver of registration fees and market exclusivity in addition to patent protection.

This is another important milestone in the development of trabedersen, a first-in-class, targeted compound based on antisense technology for the treatment of various aggressive tumors.

Highly promising preliminary efficacy data
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 preliminary clinical data are quite impressive" states the Committee on Orphan Medicinal Products (COMP) of the EMEA in its report.

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.

Median overall survival (mOS) for patients in the first schedule was 6.8 months (status Aug 2009). Moreover, one patient with recurrent advanced pancreatic cancer (after surgical resection and three chemotherapies) and liver metastases had a complete response and is still alive 45.6 months after receiving trabedersen therapy (as of Feb 2009).

The current mOS for pancreatic carcinoma patients in the first cohort of five patients in the second schedule is 13.4 months (as of Aug 2009). One patient is still alive 19 months after start of study treatment (as of April 2009).

In the same study good safety, tolerability and encouraging first efficacy data for trabedersen was observed also in patients with advanced malignant melanoma or colorectal carcinoma. Of five malignant melanoma patients treated with trabedersen, one from the first schedule showed stable disease and lived for 13.8 months.

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