EU/3/17/1833 - orphan designation for treatment of plasma cell myeloma
autologous T-cells transduced with lentiviral vector encoding an anti-SLAMF7 CD28/CD3-zeta chimeric antigen receptor
OrphanHuman
Overview
On 27 February 2017, orphan designation (EU/3/17/1833) was granted by the European Commission to Dr. Michael Hudecek, Germany, for autologous T-cells transduced with lentiviral vector encoding an anti-SLAMF7 CD28/CD3-zeta chimeric antigen receptor for the treatment of plasma cell myeloma.
Plasma cell myeloma (also called multiple myeloma) is a cancer of a type of white blood cell called plasma cells. Plasma cells originate from the bone marrow, the spongy tissue inside the large bones in the body. In plasma cell myeloma the division of plasma cells becomes out of control, resulting in abnormal, immature plasma cells multiplying and filling up the bone marrow. This interferes with the 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.
Plasma cell 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, plasma cell myeloma affected less than 4 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 206,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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 515,700,000 (Eurostat 2017).
At the time of designation, several medicines were already authorised for plasma cell myeloma in the EU. The main treatment for plasma cell myeloma was chemotherapy (medicines to treat cancer) usually combined with corticosteroids to reduce the activity of the immune system, the body's natural defences. Where chemotherapy did not work, some patients received an allogeneic stem-cell transplant (a complex procedure where the patient receives stem cells from a matched donor to help restore the bone marrow). Radiotherapy (using radiation to kill cancer cells) was used to treat pain due to bone damage and prevent further damage. Interferon alfa was sometimes used in combination with chemotherapy.
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with plasma cell myeloma. Laboratory studies showed that the medicine helped eliminate myeloma cells from the blood, spleen and bone marrow and improved survival, and its effects compared well with those of other authorised treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The plasma cells in patients with the condition produce a protein on their surface called SLAMF7. To make this medicine, T cells (a type of white blood cell that is part of the body's natural defences) are taken from the patient. They are then modified in the laboratory by a virus that carries a gene into the cells which allows them to target SLAMF7. The modified T cells are grown to increase their numbers before being given back to the patient. Once the modified T cells are returned to the patient, they are expected to recognise SLAMF7 on the cancerous plasma cells, allowing the T cells to target and kill them.
The virus used in this medicine ('lentivirus') is modified in order not to cause disease in humans.
The effects of the medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with plasma cell myeloma had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for plasma cell 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 19 January 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 T-cells transduced with lentiviral vector encoding an anti-SLAMF7 CD28/CD3-zeta chimeric antigen receptor
- Intended use
- Treatment of plasma cell myeloma
- Orphan designation status
- Positive
- EU designation number
- EU/3/17/1833
- Date of designation
- Sponsor
Dr. Michael Hudecek
Winterleitenweg 108
97204 Höchberg
Germany
Tel. +49 931 201 71091
E-mail: Hudecek_M@ukw.de
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product it 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: