Cancer Research UK secures its first orphan drug licence

Cancer Research UK

A previously shelved drug could prove effective for treating a group of rare childhood cancers, and Cancer Research UK today announced it has been granted exclusive marketing rights for the drug in these indications by the European Commission and the Food and Drug Administration (FDA).

The drug, called fenretinide, was first made in the 1970s by pharmaceutical giant Johnson & Johnson as a possible treatment for breast cancer, but the company didn’t bring 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 (0-24 years old), usually diagnosed in adolescence, in the EU each year.

A number of laboratory studies of fenretinide in ESFT cells were carried out by Dr Sue Burchill, based at Cancer Research UK’s Clinical Centre in Leeds and supported by the local children’s cancer charity, Candlelighter’s. 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.

Drugs for rare diseases are called ‘orphans’ because the small number of patients they would treat means they are often not developed by the pharmaceutical industry. Today’s decision will make it easier for Cancer Research UK’s development company, Cancer Research Technology Ltd (CRT), to attract biotech companies to partner with the charity to help develop fenretinide for clinical use. Fenretinide will be accelerated through the European Medicines Agency’s (EMEA’s) and FDA’s approval processes, and CRT and its partners will have ten and seven years to exclusively market the drug in the EU and US respectively.

The next step in the development of fenretinide is a phase II trial, which has been approved for funding by Cancer Research UK.

Dr Ian Lewis, the paediatric oncologist who will be leading the trial at St James’s University Hospital, Leeds, said: "Fenretinide has already been tested in adults and children in the first phase of clinical trials, so we know how it affects the body. 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. A young person currently diagnosed with one of these forms of tumour will be treated with a cocktail of general chemotherapy drugs, surgery or radiotherapy. Despite improvement in treatment, many young people still succumb to ESFT. If we can confirm the effectiveness of fenretinide, it could significantly improve outcomes for this rare group of cancers."

Cancer Research UK and CRT recently launched the Clinical Development Partnerships (CDP) programme to target drugs that have been ‘deprioritised’ by pharmaceutical companies. Although fenretinide has not come through the CDP programme, it highlights the possibility of finding innovative routes to take more potential cancer therapies into clinical trials.

Harpal Kumar, chief operating officer of Cancer Research UK and chief executive of CRT, said: "This is the first time that Cancer Research UK has applied for orphan drug designation, and it represents our ongoing commitment to developing new treatments for all types of cancer, no matter how rare. Without this decision, it would be difficult, once we have completed our phase II trial, to attract biotech companies to form partnerships with us, which will be vital to successfully developing fenretinide as a treatment for ESFT."

ENDS

For media enquiries please contact Michael Regnier in the Cancer Research UK press office on 020 7061 8309 or, out of hours, on 07050 264 059.

Notes to Editor

  • Fenretinide is a molecule with a similar structure to vitamin A.
  • It has recently been tested in comparison to tamoxifen for treating breast cancer in phase III clinical trials, and is being developed by other groups for use in treating neuroblastoma.
  • 5-year survival rates for children with Ewing’s sarcoma are around 60 per cent for those diagnosed with localised tumours, and around 20 per cent for those diagnosed with metastatic disease.
  • The EMEA was set up by the European Commission in 1995 to centralise drug approval in the EU. One of its committees is responsible for medicines for rare diseases - the Committee for Orphan Medicinal Products. Click here to visit EMEA's website.
  • The Food and Drug Administration is one of the US’s oldest consumer protection agencies. The FDA's mission is:
    • to promote and protect the public health by helping safe and effective products reach the market in a timely way,
    • To monitor products for continued safety after they are in use, and
    • To help the public get the accurate, science-based information needed to improve health. Click here to visit the FDA's website.
  • Orphan designation prioritises medicinal products in the approval process - applicants receive guidance from the EMEA and FDA, together with fee reductions, accelerated approval and market exclusivity.

About Cancer Research Technology Limited

Cancer Research Technology Limited (CRT) is an oncology-focused development and commercialisation company that realises cancer patient benefit from publicly- funded research worldwide. CRT works closely with leading international cancer scientists, their institutes and funding bodies to protect, develop and commercialise oncology-related discoveries. CRT is wholly owned by Cancer Research UK, the largest independent funder of cancer research in the world. Further information about CRT can be found at their website.

About Cancer Research UK

Together with its partners and supporters, Cancer Research UK's vision is to beat cancer.

  • Cancer Research UK carries out world-class research to improve understanding of the disease and find out how to prevent, diagnose and treat different kinds of cancer.
  • Cancer Research UK ensures that its findings are used to improve the lives of all cancer patients.
  • Cancer Research UK helps people to understand cancer, the progress that is being made and the choices each person can make.
  • Cancer Research UK works in partnership with others to achieve the greatest impact in the global fight against cancer.

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