Bayer Pharmaceuticals Corporation and Onyx Pharmaceuticals Inc announced that BAY 43-9006 has been granted orphan drug status for the treatment of renal cell carcinoma, by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMEA). BAY 43-9006 is being evaluated for the treatment of metastatic renal cell carcinoma, an advanced form of kidney cancer.

BAY 43-9006, a novel investigational drug candidate, is also under review for Orphan Drug designation by US FDA. Currently in worldwide phase III clinical testing, BAY 43-9006 is a novel RAF kinase and VEGFR inhibitor that is intended to prevent tumour growth by combining two important anticancer activities: inhibition of tumour cell proliferation and tumour angiogenesis. BAY 43-9006 is being co-developed by Bayer and Onyx.

The EMEA orphan drug designation provides incentives to companies that develop drugs for diseases affecting less than five in 10,000 people in the European Union (EU). The designation provides a drug EU market exclusivity, for a particular indication, against drugs with the same principal molecular structural features and which act via the same mechanism. The market exclusivity is for a 10-year period if the sponsor complies with certain EMEA specifications.

"The orphan drug designation in the EU signifies another important step for Bayer and Onyx in the development of BAY 43-9006," Susan Kelley, vice president, Oncology, Bayer Pharmaceuticals Corporation said adding, "We appreciate the EMEA's recognition of our efforts and investment to bring BAY 43-9006 forward for the potential benefit of patients with this devastating form of advanced renal cell cancer."

BAY 43-9006 is being evaluated in multiple tumour types as a single therapy and in combination with conventional chemotherapeutics. In preclinical models, BAY 43-9006 has shown anticancer activity in a number of tumour types and has inhibited tumour cell proliferation by targeting the RAF/MEK/ERK signalling pathway at the level of RAF kinase. BAY 43-9006 also exerted an anti-angiogenic effect by targeting the receptor tyrosine kinases VEGFR-2 and PDGFR and their associated signalling cascades.

Bayer and Onyx have initiated an international, multi-centre phase III trial to further evaluate the safety and efficacy of BAY 43-9006 in the treatment of advanced renal cell carcinoma (RCC). In the trial, approximately 800 people will participate at sites worldwide. The primary objective of the randomized study will be to confirm the early indicators of clinical activity of BAY 43-9006 in RCC, using improvement in survival as the primary endpoint for assessment of clinical benefit.

Renal cell carcinoma is the most common form of kidney cancer. Nearly 190,000 people worldwide (about 81,000 in Europe) are diagnosed with renal cell carcinoma each year, and more than 91,000 of them (about 39,000 in Europe) die from the disease annually.