EU/3/10/803 - orphan designation for treatment of gastric cancer

Chimeric monoclonal antibody against claudin-18 splice variant 2

Overview

On 26 November 2010, orphan designation (EU/3/10/803) was granted by the European Commission to Ganymed Pharmaceuticals AG, Germany, for chimeric monoclonal antibody against claudin-18 splice variant 2 for the treatment of gastric cancer.

In May 2017 sponsor, Ganymed Pharmaceuticals AG changed name to Ganymed Pharmaceuticals AG GmbH.

The sponsorship was transferred to Astellas Pharma Europe B.V., The Netherlands, in March 2018.

What is gastric cancer?

Gastric cancer is a cancer that starts in the stomach, generally in the glandular cells lining the inside of the stomach. Gastric cancer is often detected late as the early signs of the disease are the same as those of less serious stomach conditions (heartburns, gas, excessive belching). At a later stage, gastric cancer causes unexplained weight loss, loss of appetite and general decline in health. Bleeding can occur, leading to anaemia (low red blood cell counts). Men are about twice as likely to develop the disease as women.

Gastric cancer is a serious and life-threatening illness that is associated with shortened life expectancy.

What is the estimated number of patients affected by the condition?

At the time of designation, gastric cancer affected approximately 3 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 152,000 people, and is below the threshold 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 506,500,000 (Eurostat 2010).

What treatments are available?

At the time of designation, patients with gastric cancer were treated first with surgery to remove part of or the whole stomach. Chemotherapy (medicines to treat cancer) was generally used after surgery. Many chemotherapy medicines were authorised in the EU for use in gastric cancer, such as docetaxel, doxorubicin, capecitabine, carmustine, epirubicin, 5-fluorouracil, mitomycin C and trastuzumab. They were often used in combination.

The sponsor has provided sufficient information to show that chimeric monoclonal antibody against claudin-18 splice variant 2 might be of significant benefit for patients with gastric cancer because it works in a different way to existing treatments and early studies in experimental models show that it might improve the treatment of patients with this condition. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

Claudin-18 splice variant 2 is a protein found in the glands lining the inside of the stomach, where it helps the gastric cells to stick to each other. In patients with gastric cancer, this protein is much more widespread and is thought to be involved in the survival and spread of the cancer cells.

The medicine is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to a part of the claudin-18 splice variant 2 protein in the gastric cancer cells. By attaching to this protein, this medicine is expected to block the growth of cancer cells, slowing down the spread of the cancer.

What is the stage of development of this medicine?

The effects of chimeric monoclonal antibody against claudin-18 splice variant 2 have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with this medicine in patients with gastric cancer were ongoing.

At the time of submission, the medicinal product was not authorised anywhere in the EU for gastric 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 9 September 2010 recommending the granting of this designation

Opinions on orphan medicinal product designations are based on the following three criteria

• 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 European Union) 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.

EU/3/10/803: Public summary of opinion on orphan designation chimeric monoclonal antibody against claudin-18 splice variant 2 for the treatment of gastric cancer

AdoptedReference Number: EMA/579374/2010

Key facts

• Active substance:
  Chimeric monoclonal antibody against claudin-18 splice variant 2
• Intended use:
  Treatment of gastric cancer
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/10/803
• Date of designation:
  26/11/2010
• Sponsor:

  Astellas Pharma Europe B.V.; Sylviusweg 62; 2333 BE Leiden; The Netherlands; Tel. [phone]; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.