Cancer Research UK says that it has been granted exclusive marketing rights in Europe and the USA for a previously-shelved drug, fenretinide, and in doing so has secured its first orphan drug licence.
Fenretinide was first developed in the 1970s by Johnson & Johnson as a possible treatment for breast cancer, but never made it to market. Now out of patent, fenretinide has been studied by research groups in relation to various types of cancer, including the Ewing’s sarcoma family of tumours (ESFT), a group of rare cancers that affect around one in every million young people, usually diagnosed in adolescence, in the EU each year.
The charity noted that a number of laboratory studies of fenretinide in ESFT cells were carried out by Dr Sue Burchill of the University of Leeds, and “her promising results” convinced Cancer Research UK to apply for orphan designation for the use of fenretinide in the treatment of soft tissue sarcomas and malignant bone tumours – two categories of disease in the ESFT group.
Getting orphan status will make it easier for Cancer Research UK’s development company, Cancer Research Technology , to attract biotechnology companies to partner with the charity to help develop fenretinide, which will be accelerated through the European Medicines Agency’s and the US Food and Drug Administration’s approval processes. It was noted that CRT and its partners will have ten and seven years to exclusively market the drug in the EU and USA respectively.
A Phase II trial of fenretinide is being planned and Dr Ian Lewis, the paediatric oncologist who will be leading the study at St James’s University Hospital, Leeds, noted that the drug has already been tested in adults and children in the first phase “ so we know how it affects the body.” He added that “we can safely move to Phase II trials, and see if fenretinide lives up to the promise it has shown in the laboratory for Ewing’s sarcoma and related cancers.”