EU/3/04/252 - orphan designation for treatment of chronic myeloid leukaemia

Val-Leu-Gln-Glu-Leu-Asn-Val-Thr-Val (Pr1 nanopeptide, sequence 169-177, of proteinase 3)

Overview

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in March 2009 on request of the Sponsor.

On 21 December 2004, orphan designation (EU/3/04/252) was granted by the European Commission to Accelsiors CRO & Consultancy Services GmbH, Germany, for Val-Leu-Gln-Glu-Leu-Asn-Val- Thr-Val (Pr1 nanopeptide, sequence 169-177, of proteinase 3) for the treatment of chronic myeloid leukaemia.

What is chronic myeloid leukaemia?

Chronic myeloid leukaemia is a disease in which cancer cells are found in the blood and in the bone marrow. The bone marrow is the spongy tissue inside the large bones in the body. Normally, the bone marrow makes cells called “blasts” that mature into several different types of blood cells that have specific functions in the body. These include red cells, white cells and platelets. Red blood cells carry oxygen and other materials to all tissues of the body. White blood cells fight infection. Platelets make the blood clot. When leukaemia develops, the bone marrow produces large numbers of abnormal blood cells. There are several types of leukaemias. In myeloid leukaemia blasts that are developing into white blood cells called granulocytes, are affected. The blasts do not mature and become too many. These blast cells are then found in the blood and also accumulate in the bone marrow. The disease can develop very slowly, which is why it is called “chronic” myeloid leukaemia. Chronic myeloid leukaemia is life-threatening

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

At the time of designation, chronic myeloid leukaemia affected approximately 0.4 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 18,000 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 25), Norway, Iceland and Liechtenstein. This represents a population of 459,700,000 (Eurostat 2004).

What treatments are available?

Treatment for leukaemia is complex and depends on a number of factors including the type of leukaemia, the extent of the disease and whether the leukaemia has been treated before. It also depends on the age, the symptoms, and the general health of the patient. At the time of submission of the application for orphan drug designation authorised treatments of chronic myeloid leukaemia included chemotherapy agents (using drugs to kill cancer cells) and immunotherapy agents (using drugs that stimulate the body's own immune system to kill cancer cells). Sometimes a combination of immunotherapy and chemotherapies may be used. Another product is also used which blocks (inhibits) growth signals within cancer cells and prevents a series of chemical reactions that cause the cell to grow and divide thus stopping cancer cells growth. Bone marrow transplantation is also used.

Val-Leu-Gln-Glu-Leu-Asn-Val-Thr-Val (Pr1 nanopeptide, sequence 169-177, of proteinase 3) could be of potential significant benefit for the treatment of chronic myeloid leukaemia because it may act in a different way and it might improve the long-term outcome of patients. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

Val-Leu-Gln-Glu-Leu-Asn-Val-Thr-Val (Pr1 nanopeptide, sequence 169-177, of proteinase 3) is a peptide, which is a part of a protein. It is similar to a part of a protein, present on the surface of certain cells, such as cancer cells. It is expected to work as a vaccine, thus upon administration of this peptide, the body's defense system (immune system) will recognize this peptide as a foreign body and react against this peptide. Since part of this foreign body is similar to parts of the protein on the cancer cells, it is expected that the immune system will not only recognize the vaccine as foreign, but also the cancer cells. The body's immune system could then kill these cancer cells.

What is the stage of development of this medicine?

The effects of Val-Leu-Gln-Glu-Leu-Asn-Val-Thr-Val (Pr1 nanopeptide, sequence 169-177, of proteinase 3) were evaluated in experimental models.

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

Val-Leu-Gln-Glu-Leu-Asn-Val-Thr-Val (Pr1 nanopeptide, sequence 169-177, of proteinase 3) was not marketed anywhere worldwide for the treatment of chronic myeloid leukaemia or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

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/3/04/252: Public summary of positive opinion for orphan designation of Val-Leu-Gln-Glu-Leu-Asn-Val-Thr-Val (Pr1 nanopeptide, sequence 169-177, of proteinase 3) for the treatment of chronic myeloid leukaemia

Adopted Reference Number: EMEA/COMP/152652/2004

Key facts

• Active substance:
  Val-Leu-Gln-Glu-Leu-Asn-Val-Thr-Val (Pr1 nanopeptide, sequence 169-177, of proteinase 3)
• Intended use:
  Treatment of chronic myeloid leukaemia
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/04/252
• Date of designation:
  21/12/2004
• Sponsor:

  Accelsiors CRO & Consultancy Services GmbH; Hammersbacher Fussweg 24; 82467 Garmisch-Partenkirchen; Germany; Telephone: [phone]; Telefax: [phone]; E-mail: [e-mail]