EU/3/14/1395 - orphan designation for treatment of acute myeloid leukaemia

allogeneic ex vivo-generated natural killer cells from CD34+ umbilical cord blood progenitor cells

Overview

On 16 December 2014, orphan designation (EU/3/14/1395) was granted by the European Commission to IPD-Therapeutics BV, the Netherlands, for allogeneic ex vivo-generated natural killer cells from CD34+ umbilical cord blood progenitor cells for the treatment of 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 blood cells, causing bleeding episodes, blood clots and 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 approximately 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 51,000 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 28), Norway, Iceland and Liechtenstein. This represents a population of 511,100,000 (Eurostat 2014).

What treatments are available?

The 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 this medicine might be of significant benefit for patients with AML because early studies in experimental models suggest that it can improve responses in patients with this condition when used together with other treatments. 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?

This medicine is made up of certain cells called natural–killer (NK) cells that belong to the immune system (the body's natural defences) and that act against infections or tumours. To produce these NK cells, immature blood cells (called CD34+ cells) are taken from donor blood obtained from the umbilical cord that connects a baby to the placenta. These cells are able to develop into different cell types. They are grown outside the body and encouraged to develop into NK cells. When injected into the patient, the NK cells are expected to target and destroy AML cells, thereby slowing down the growth of the cancer.

What is the stage of development of this medicine?

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicine 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 13 November 2014 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.

EU/3/14/1395: Public summary of opinion on orphan designation: Allogeneic ex vivo-generated natural killer cells from CD34+ umbilical cord blood progenitor cells for the treatment of acute myeloid leukaemia

Adopted Reference Number: EMA/COMP/730059/2014

Key facts

• Active substance:
  allogeneic ex vivo-generated natural killer cells from CD34+ umbilical cord blood progenitor cells
• Intended use:
  Treatment of acute myeloid leukaemia
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/14/1395
• Date of designation:
  16/12/2014
• Sponsor:

  IPD-Therapeutics BV; Pivot Park RE1134; Kloosterstraat 9; 5349 AB Oss; 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.