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Orphan designation

On 9 October 2009, orphan designation (EU/3/09/673) was granted by the European Commission to Affimed Therapeutics AG, Germany, for recombinant antibody construct against human CD30 and CD16A for the treatment of Hodgkin’s lymphoma.

In January 2016, Affimed Therapeutics AG changed name to Affimed GmbH.

What is Hodgkin’s lymphoma?

Hodgkin’s lymphoma is a type of cancer of the lymphatic system, a network of vessels that transport lymph from tissues through the lymph nodes and into the bloodstream. Because the lymphatic system is found throughout the body, the cancer can begin in almost any part of the body.

In Hodgkin’s lymphoma, the lymphatic cancer cells multiply too quickly and live for too long, so there are too many of them in a lymph node. Sometimes these cells spread through the lymphatic system to other lymph nodes and they may also enter the bloodstream which carries them to various organs, forming new tumours. When the disease progresses, patients may develop serious infections and sepsis (generalised spreading of infection through the blood).

Many people with Hodgkin’s lymphoma can be cured if the disease is found and treated early.

However, despite the available treatments, Hodgkin’s lymphoma remains a serious and life-threatening disease in some patients because of its complications.

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

At the time of designation, Hodgkin’s lymphoma affected approximately 1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of 50,000 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 Hodgkin’s lymphoma in the EU. The main treatments for Hodgkin’s lymphoma included chemotherapy (medicines to treat cancer) and radiotherapy (treatment with radiation). Autologous bone marrow transplantation was also used when patients had not responded to treatment or when the disease had come back after treatment. This is a complex procedure where the bone marrow of the patient is destroyed and replaced with healthy bone marrow previously obtained from the same patient.

The sponsor has provided sufficient information to show that recombinant antibody construct against human CD30 and CD16A might be of significant benefit for patients with Hodgkin’s lymphoma because it works in a different way to existing treatments. Early studies indicate that it might represent an alternative way to treat Hodgkin’s lymphoma, in particular in patients who cannot receive or do not respond to standard chemotherapy. These assumptions 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?

Recombinant antibody construct against human CD30 and CD16A is a protein that is made up of two antibody-like components, each of which is designed to recognise and attach to a specific structure (antigen):

  • CD30, a receptor that is found in large amounts on the surface of Hodgkin’s lymphoma cells;
  • CD16A, a receptor that is found on the surface of cells of the immune system (the body’s natural defences) called natural killer cells and macrophages.

This medicine is expected to attach simultaneously to Hodgkin’s lymphoma cells through CD30, and natural killer cells and macrophages through CD16A. By attaching to CD16A, this medicine is expected to stimulate the natural killer cells and macrophages to kill the Hodgkin’s lymphoma cells to which they are attached.

What is the stage of development of this medicine?

The effects of recombinant antibody construct against human CD30 and CD16A have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials in patients with Hodgkin’s lymphoma had been started.

At the time of submission, this medicine was not authorised anywhere in the EU for Hodgkin’s lymphoma 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 July 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.

Key facts

Product details for <p>Recombinant antibody construct against human CD30 and CD16A</p>
Active substanceRecombinant antibody construct against human CD30 and CD16A
Medicine Name
Disease/conditionTreatment of Hodgkin’s lymphoma
Date of decision09/10/2009
Orphan decision numberEU/3/09/673

Review of designation

Sponsor’s contact details

Affimed GmbH
Im Neuenheimer Feld 582
69120 Heidelberg
Tel. +49 6221 65307 65
Fax +49 6221 65307 49

Patients’ organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.