to-BBB receives positive opinion for Orphan Drug Designation

16-Jun-2010 - Netherlands

to-BBB has received a positive opinion from the Committee for Orphan Medicinal Products (COMP) on the orphan medicinal product designation for the company’s brain cancer lead candidate 2B3-101.

As a proprietary brain-targeted version of the marketed product Caelyx®/Doxil® (PEG-liposomal doxorubicin), 2B3-101 uses glutathione (GSH) to safely enhance the delivery of doxorubicin across the blood-brain barrier. The therapeutic benefit and predictable safety profile of 2B3-101 has been successfully demonstrated in pre-clinical studies and the product is ready to enter clinical trials end of 2010. The product can be developed for multiple brain cancer indications and to-BBB considers development for brain metastases of breast cancer as well as glioma.

The positive opinion for 2B3-101 by the COMP has specifically been granted for the treatment of glioma, which affects less than 5 per 10,000 people in the European Community. The low prevalence of this life-threatening condition makes drug development otherwise unappealing, especially for emerging biotech companies such as to-BBB. However, when the European Commission decides to grant the orphan drug status following the positive opinion of the COMP, to-BBB would receive additional market exclusivity for a period of ten years following approval of its drug candidate for this indication in Europe. Thereby the Orphan Drug Designation will increase the likelihood that 2B3-101 will become available for glioma patients.

“We thank the COMP for their positive opinion,” says Willem van Weperen, CEO of to-BBB. “We are excited about the potential of 2B3-101 and, supported by the benefits of the Orphan Drug Designation, we will continue to execute our strategy to develop this therapy for patients with life-threatening brain cancer.”

The Commission typically grants orphan drug status following a positive opinion from the COMP to a medicinal product intended for the diagnosis, prevention or treatment of a life-threatening or a chronically debilitating condition when the application for designation is submitted.

https://www.bionity.com/en/news/118957/to-bbb-receives-positive-opinion-for-orphan-drug-designation.html