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Orphan designation

On 8 February 2013, orphan designation (EU/3/13/1106) was granted by the European Commission to ERYtech Pharma S.A., France, for L-asparaginase encapsulated in erythrocytes for the treatment acute myeloid leukaemia.

What is acute myeloid leukaemia?

Acute myeloid leukaemia (AML) is a cancer of the white blood cells (cells that fight against infections). In patients with AML, the bone marrow (the spongy tissue inside the large bones, where blood cells are produced) produces large numbers of abnormal, immature white blood cells. These abnormal cells quickly build up in large numbers in the bone marrow and are found in the blood.

AML is a long-term debilitating and life-threatening disease because these abnormal immature cells take the place of the normal white blood cells, reducing the patient’s ability to fight infections.

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

At the time of designation, AML affected not more than 1.2 in 10,000 people in the European Union (EU). This was equivalent to a total of 61,000 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 27), Norway, Iceland and Liechtenstein. This represents a population of 509,000,000 (Eurostat 2013).

What treatments are available?

Treatment for AML is complex and depends on a number of factors including the extent of the disease, whether it has been treated before, and the patient’s age, symptoms and general state of health. At the time of designation, the main treatments for AML were chemotherapy (medicines to treat cancer) and haematopoietic (blood) stem-cell transplantation (a complex procedure where the patient receives stem cells from a matched donor to help restore the bone marrow).

The sponsor has provided sufficient information to show that L-asparaginase encapsulated in erythrocytes might be of significant benefit for patients with AML because early studies show that it might improve treatment of patients when it is used in combination with another chemotherapy medicine called cytarabine compared with patients treated with cytarabine alone. 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?

L-asparaginase is an enzyme that breaks down the substance L-asparagine, which is required for cell growth. Certain cancer cells, such as the cancerous cells in AML, cannot make L-asparagine, so they need to take it up from the blood in order to grow. By reducing the levels of L-asparagine in the blood, this medicine is expected to deprive the cancerous AML cells of their supply of L-asparagine, causing them to die. L-asparaginase has already been used for the treatment of acute lymphoblastic leukaemia (another cancer of the white blood cells) since the 1970s. 

This medicine is made up of erythrocytes (red blood cells) that have been loaded with L-asparaginase so that the L-asparaginase is ‘encapsulated’ (contained) within the erythrocytes. The erythrocytes reduce the exposure of L-asparaginase to the immune system (the body’s natural defences). This results in the immune system producing fewer antibodies against L-asparaginase, which could otherwise cause side effects such as allergic reactions. 

The erythrocytes also form tiny compartments where the breakdown of L-asparagine can take place. Together, these properties are expected to increase how long L-asparaginase remains active in the body and to allow a lower dose of the enzyme to be used for the same anticancer effect as the free enzyme.

What is the stage of development of this medicine?

The effects of L-asparaginase encapsulated in erythrocytes have been evaluated in experimental models. 

At the time of submission of the application for orphan designation, no clinical trials with L-asparaginase encapsulated in erythrocytes in patients with AML were ongoing. 

At the time of submission, the medicine was not authorised anywhere in the EU for AML 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 January 2013 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 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.

Key facts

Product details for <p>L-asparaginase encapsulated in erythrocytes</p>
Active substanceL-asparaginase encapsulated in erythrocytes
Medicine Name
Disease/conditionTreatment acute myeloid leukaemia
Date of decision08/02/2013
Orphan decision numberEU/3/13/1106

Review of designation

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

Sponsor’s contact details

ERYtech Pharma S.A
60, avenue Rockefeller
Bâtiment Adenine
69008 Lyon
Telephone: +33 478 744 438
Telefax: +33 478 755 629

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.