EU/3/17/1918 - orphan designation for treatment of multiple myeloma
autologous ex-vivo-expanded peripheral polyclonal lymphocytes enriched in activated natural killer cells
OrphanHuman
Overview
On 16 October 2017, orphan designation (EU/3/17/1918) was granted by the European Commission to CellProtect Nordic Pharmaceuticals AB, Sweden, for autologous ex vivo expanded peripheral polyclonal lymphocytes enriched in activated natural killer cells for the treatment of multiple myeloma.
Multiple myeloma (also called plasma cell myeloma) is a cancer of a type of white blood cell called plasma cells. Plasma cells are produced in the bone marrow, the spongy tissue inside the large bones in the body. In multiple myeloma, the division of plasma cells becomes uncontrolled, resulting in abnormal, immature plasma cells multiplying and filling up the bone marrow. This interferes with production of normal white blood cells, red blood cells and platelets (components that help the blood to clot), leading to complications such as anaemia (low red blood cell counts), bone pain and fractures, raised blood calcium levels and kidney disease.
Multiple myeloma is a debilitating and life-threatening disease particularly because it disrupts the normal functioning of the bone marrow, damages the bones and causes kidney failure.
At the time of designation, multiple myeloma affected no more than 4.3 in 10,000 people in the European Union (EU). This was equivalent to no more than 222,000 people1, 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).
1Disclaimer: 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 515,700,000 (Eurostat 2017).
At the time of designation, several medicines were authorised for multiple myeloma in the EU. The main treatment for multiple myeloma was chemotherapy (medicines to treat cancer) usually combined with corticosteroids to reduce the activity of the immune system, the body's natural defences. After chemotherapy patients received a stem-cell transplant if they were considered suitable for it. Stem-cell transplantation is a procedure where the patient's bone marrow is replaced with stem cells to form new bone marrow that produces healthy blood cells.
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with multiple myeloma. Early clinical data showed that the medicine was effective in patients with multiple myeloma who were to undergo a stem-cell transplant. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The medicine is made up of a type of white blood cells called natural-killer (NK) cells that belong to the immune system (the body's natural defences) and that act against infections or cancers. To produce the medicine, NK cells are taken from the patient's own blood, grown outside the body to increase their number and activated to increase their anticancer activity. When injected into the patient, the NK cells are expected to destroy cancer cells, thereby slowing down the growth of the cancer.
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 multiple myeloma were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for multiple myeloma 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 7 September 2017 recommending the granting of this designation.
- 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
- Active substance
- autologous ex-vivo-expanded peripheral polyclonal lymphocytes enriched in activated natural killer cells
- Intended use
- Treatment of multiple myeloma
- Orphan designation status
- Positive
- EU designation number
- EU/3/17/1918
- Date of designation
- Sponsor
CellProtect Nordic Pharmaceuticals AB
Hälsovägen 7
SE 141 57 Huddinge
Sweden
Tel. + 46 70 230 8002
E-mail: info@cellprotect.se
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
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.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: