AstraZeneca has received positive opinion from the Committee for Orphan Medicinal Products (COMP) recommending orphan drug designation for ZD6474 (Zactima) for the treatment of patients with medullary thyroid cancer in the European Union (EU).

According to the company release, final adoption of the opinion is expected from the European Commission in early 2006. ZD6474 is a once-daily oral therapy that selectively targets key cell signalling pathways involved in tumour growth and spread including vascular endothelial growth factor (VEGF) receptor signalling and epidermal growth factor (EGF) receptor signalling. ZD6474 also inhibits RET kinase, which drives the growth and survival of certain tumours, and is believed to be an important pathway in medullary thyroid cancer.

"The COMP's recommendation adds further momentum behind the development of ZD6474 and represents a major step forward in the EU orphan drug designation process. EU orphan drug designation would offer hope for patients in Europe with medullary thyroid cancer, where treatment options are limited and the prognosis is poor," said Peter Langmuir, senior medical science director, AstraZeneca.

A recent presentation of preliminary results from a phase II study during the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in November 2005 showed promising clinical data with ZD6474 (Zactima) in patients with hereditary medullary thyroid cancer. A phase II study evaluating the anti-tumour efficacy and safety of ZD6474 in patients with advanced hereditary medullary thyroid cancer is currently ongoing and continues to recruit patients.

Thyroid cancer affects 240,989 individuals in Europe. Medullary thyroid cancer is a specific form of the disease accounting for 5-8 per cent of the 25,000 new cases of thyroid cancer in Europe each year.

ZD6474 received US FDA orphan drug designation in October 2005 for patients with follicular, medullary, anaplastic, and locally advanced and metastatic papillary thyroid cancer, the first oncology orphan drug designation AstraZeneca has received.