EU/3/16/1628

Oesophageal cancer is cancer of the oesophagus (the tube that connects the mouth to the stomach). It usually starts in the cells lining the oesophagus, and can spread to other parts of the body. Oesophageal cancer is often detected late because in the early stage of the disease patients do not have noticeable symptoms. The cancer is usually diagnosed in people aged over 60 years, and men are more likely to develop the disease than women.

Oesophageal cancer is a life-threatening disease because of its serious complications such as dysphagia (difficulty swallowing), bleeding and weight loss that lead to poor overall survival.

At the time of designation, oesophageal cancer affected approximately 0.8 in 10,000 people in the European Union (EU). This was equivalent to a total of around 41,000 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 28), Norway, Iceland and Liechtenstein. This represents a population of 513,700,000 (Eurostat 2016).

The choice of treatment for oesophageal cancer depends on where in the oesophagus the cancer is located, and how advanced it is. At the time of orphan designation, the main treatments were surgery, radiotherapy (treatment with radiation) and chemotherapy (medicines to treat cancer).

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with oesophageal cancer because early laboratory studies showed that when used with cisplatin and fluorouracil it further reduced tumour growth. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Because of the irregular growth of blood vessels in cancers, some parts of the cancer have low levels of oxygen (hypoxia). Lack of oxygen makes cancer cells become resistant to cancer treatments.

This medicine contains haemoglobin, the protein in red blood cells that carries oxygen around the body. It is expected to reach hypoxic cancer cells and to deliver oxygen to them. Since hypoxic cells are unable to cope with large amounts of oxygen, the increased oxygen levels are expected to reduce resistance to cancer treatment and lead to death of the cancer cells, thus slowing down the growth of the cancer.

The effects of this medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with oesophageal cancer had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for oesophageal cancer 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 18 February 2016 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.