FDA and EMEA grant trabedersen orphan drug status
German biopharmaceutical company Antisense Pharma has received orphan drug designation from the European Medicines Agency (EMEA) and the US Food and Drug Administration (FDA) for its investigational drug trabedersen in the treatment of pancreatic cancer.
German biopharmaceutical company Antisense Pharma has received orphan drug designation from the European Medicines Agency (EMEA) and the US Food and Drug Administration (FDA) for its investigational drug trabedersen in the treatment of pancreatic cancer.
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 people in the European Community and fewer than 200,000 people in the US, in cases where no other satisfactory therapy exists or the medicinal product is expected to provide significant benefit over existing therapies.
The designation is meant to encourage pharmaceutical companies to develop drugs for diseases that meet the above criteria by providing scientific advice, a reduction or waiver of registration fees and market exclusivity in addition to patent protection.
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 company says.
Antisense Pharma may now sell trabedersen without competition for seven years in the US and for ten years in the EU following market approval.
"Based on the results of several clinical studies, we expect trabedersen significantly to 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," said Dr Karl-Hermann Schlingensiepen, ceo of Antisense Pharma.