EU/3/10/844: Orphan designation for the treatment of renal-cell carcinoma

Renal-cell carcinoma is a type of kidney cancer that originates in the cells lining the kidney tubules. These are small tubes that filter waste products out from the blood and make urine. Signs of renal-cell carcinoma are difficult to detect in the early stages of the disease, and about half of the patients are diagnosed when the cancer has spread around the kidney or to other parts of the body.

Renal-cell carcinoma is more common in men than in women. It is a life-threatening disease that is associated with poor long-term survival.

At the time of designation, renal-cell carcinoma affected approximately 4 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 202,520 people, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 27), Norway, Iceland and Liechtenstein. This represents a population of 506,300,000 (Eurostat 2011).

At the time of designation, several medicines were authorised in the EU for the treatment of renal-cell carcinoma. The main treatment was surgery, which was combined with radiotherapy (treatment with radiation) and chemotherapy (medicines to treat cancer) when the cancer had spread outside of the kidneys.

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with renal cell carcinoma as it could, through its mechanism of action, complement medicines currently available, as shown in early studies. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Axitinib is expected to work by blocking the activity of 'vascular-endothelial-growth-factor' (VEGF) receptors. These receptors, which can be found on the surface of cancer cells, are involved in the development of new blood vessels that supply the tumours. By blocking VEGF receptors, this medicine is expected to slow down the growth of cancer cells by reducing their blood supply.

The effects of axitinib have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with axitinib in patients with renal-cell carcinoma were ongoing.

At the time of submission, axitinib was not authorised anywhere in the EU for renal-cell carcinoma or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 December 2010 recommending the granting of this designation.

• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.