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FDA and EMEA grant orphan drug designation for Antisense Pharma's investigational drug trabedersen in pancreatic carcinoma

Orphan drug designation ensures market exclusivity for seven to ten years after market approval.

The biopharmaceutical company Antisense Pharma GmbH announced recently 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 2002, in the treatment of high-grade gliomas.

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. 23 patients received single agent trabedersen intravenously as second-, third-, or fourth-line treatment either in a seven-day on/seven-day off or a four-day on/ten-day off schedule.

Median overall survival (mOS) for patients in the first schedule was 6.8 months (status Aug 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 were observed 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.

’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‘, says Dr. Karl-Hermann Schlingensiepen, Chief Executive Officer of Antisense Pharma.

Pancreatic carcinoma has one of the highest mortality rates of all cancers: worldwide, pancreatic cancer causes 227,000 deaths annually and is the eighth most common cause of death from cancer [1]. Current therapies comprise surgery, radiation and/or chemotherapy. Despite recent advances, the prognosis for pancreatic cancer patients is still poor.

Trabedersen is a targeted antisense compound (a phosphorothioate oligodeoxynucleotide) designed to selectively downregulate the production of a protein known as transforming growth factor-beta 2 (TGF-ß2) at the translational level [2,3]. Various aggressive tumours such as high-grade gliomas, advanced pancreatic cancer, malignant melanoma and advanced colorectal cancer cells produce an excessive amount of TGF-ß2, which has a crucial role in tumour progression (proliferation, angiogenesis and metastasis) and acts as a shield that protects the tumour from the body’s immune system [2,3,4]. By inhibiting TGF-ß2, trabedersen has multiple antitumoural effects: it hinders tumour progression, angiogenesis and metastasis [2,5]. In addition, trabedersen restores the body’s immune system by breaking down the protective shield so that the immune system can recognize and destroy the tumour cells.

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 (approximately 250,000 people in total) and fewer than 200,000 people in the United States, in the case that no other satisfactory therapy exists or the medicinal product is expected to provide a substantial benefit over existing therapies.


1 Parkin, D.M. (2005) CA Cancer. J. Clin. 55, 74–108
2 Schlingensiepen, K.H. et al. (2006) Targeted tumor therapy with the TGF-beta2 antisense compound AP 12009. Cytokine Growth Factor Rev. 17, 129–139
3 Tsamandas, A.C. et al. (2004) The potential role of TGFbeta1, TGFbeta2 and TGFbeta3 protein expression in colorectal carcinomas. Correlation with classic histopathologic factors and patient survival. Strahlenther Onkol. 180, 201–208
4 Kouvidou, C. et al. (2006) Expression of Smad4 and TGF-beta2 in colorectal carcinoma. Anticancer Res. 26, 2901–2907
5 Schlingensiepen, R. et al. (2005) Oligonucleotides 15, 94–104

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