On 28 February 2020, orphan designation EU/3/20/2252 was granted by the European Commission to Janssen-Cilag International N.V., Belgium, for autologous human T cells genetically modified ex-vivo with a lentiviral vector encoding a chimeric antigen receptor for B-cell maturation antigen (also known as JNJ-68284528 or LCAR-B38M CAR-T cells) for the treatment of multiple myeloma.
This medicine is now known as ciltacabtagene autoleucel.
The medicinal product has been authorised in the EU as Carvykti since 25 May 2022.
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 approximately 4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 208,000 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 519, 200,000 (Eurostat 2020).
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. This is because clinical data in patients who had previously received other types of treatment showed that the medicine was effective in many patients and a high number of them had no signs of the disease after treatment (complete response). 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 the patient’s own T cells (a type of white blood cell) that have been modified genetically in the laboratory so that they make a protein called chimeric antigen receptor (CAR). With this receptor on their surface, the modified cells, called CAR-T cells, can attach to a target on the surface of plasma cells called B-cell maturation antigen (BCMA). When the medicine is given to the patient, the modified T cells are expected to attach to and kill the abnormal plasma cells, thereby helping to clear the cancer from the body.
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, this medicine was not authorised anywhere in the EU for the treatment of multiple myeloma. Orphan designation had been granted in the United States for the treatment of this condition.
In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 22 January 2020, recommending the granting of this designation.
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.
Janssen-Cilag International N.V.
Turnhoutseweg 30
2340 Beerse
Antwerp
Belgium
Tel: +31621385718
E-mail: nreymond@its.jnj.com
The Committee for Orphan Medicinal Products reviewed the orphan designation of Carvykti at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.
More information is available in the Carvykti : Orphan Maintenance Assessment Report (initial authorisation).
| Date | Update |
|---|---|
| May 2022 | The medicinal product has been authorised in the EU as Carvykti since 25 May 2022. |
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: