EU/3/09/644 - orphan designation for treatment of neuroblastoma

Murine monoclonal antibody to GD2

Overview

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in January 2011 on request of the Sponsor.

On 12 June 2009, orphan designation (EU/3/09/644) was granted by the European Commission to United Therapeutics Europe Ltd, United Kingdom, for murine monoclonal antibody to GD2 for the treatment of neuroblastoma.

What is neuroblastoma?

Neuroblastoma is the most common solid tumour outside the brain in children. It is often present at birth but is usually diagnosed later when the cancer has spread to other parts of the body and the child begins to show symptoms of the disease. Neuroblastoma is a cancer of specialised nerve cells in the abdomen (tummy) and manifests itself as a lump in the abdomen or around the spine. Other symptoms may include weakness, bone pain, loss of appetite and fever.
Neuroblastoma is a life-threatening disease of childhood that leads to poor long-term survival.

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

At the time of designation, neuroblastoma affected approximately 1.1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 56,000 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 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 504,800,000 (Eurostat 2009).

What treatments are available?

At the time of designation, several medicines were authorised for the treatment of neuroblastoma in the EU. Treatments for neuroblastoma included surgery, radiotherapy (using radiation to kill the cancer cells) and chemotherapy (medicines to treat cancer).

The sponsor has provided sufficient information to show that murine monoclonal antibody to GD2 might be of significant benefit for patients with neuroblastoma because it works in a different way to existing medicines and might represent an alternative way to treat this disease. 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?

Murine monoclonal antibody to GD2 is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to a specific structure (an antigen) called GD2. GD2 is a substance that is present in high amounts on the surface of neuroblastoma cells, but not normal cells. The way this medicine works is not fully understood, but studies have shown that in the presence of the complement system, which is part of the body's defence system, the neuroblastoma cells to which the medicine is attached die.

What is the stage of development of this medicine?

The effects of murine monoclonal antibody to GD2 have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with neuroblastoma were ongoing.

At the time of submission, murine monoclonal antibody to GD2 was not authorised anywhere in the EU for neuroblastoma. Orphan designation of this medicine had been granted in the United States of America for neuroblastoma.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 5 May 2009 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 Community) 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/3/09/644: Public summary of positive opinion for orphan designation of murine monoclonal antibody to GD2 for the treatment of neuroblastoma

Adopted Reference Number: EMEA/COMP/292651/2009 Rev. 1

Key facts

• Active substance:
  Murine monoclonal antibody to GD2
• Intended use:
  Treatment of neuroblastoma
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/09/644
• Date of designation:
  12/06/2009
• Sponsor:

  United Therapeutics Europe Ltd; Unither House; Curfew Bell Road; Chertsey, Surrey; KT16 9FG; United Kingdom; Telephone: [phone]; Telefax: [phone]; E-mail: [e-mail]