EU/3/13/1153 - orphan designation for treatment of plasma-cell myeloma
Daratumumab
OrphanHuman
Overview
On 17 July 2013, orphan designation (EU/3/13/1153) was granted by the European Commission to Janssen-Cilag International N.V., Belgium, for daratumumab for the treatment of plasma-cell myeloma.
Update: daratumumab has been authorised in the EU as Darzalex since 20 May 2016.
More information on Darzalex can be found in the European public assessment report (EPAR) on the Agency's website.
Plasma-cell myeloma is a cancer of a type of white blood cells 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 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 because it disrupts the normal functioning of the bone marrow, leads to bone lesions and causes kidney failure.
At the time of designation, plasma cell myeloma affected approximately 1.75 in 10,000 people in the European Union (EU). This was equivalent to a total of around 90,000 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. At the time of designation, this represented a population of 512,200,000 (Eurostat 2013).
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 steroids 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 and weakened bones. Interferon alfa was sometimes used in combination with chemotherapy.
The sponsor has provided sufficient information to show that daratumumab might be of significant benefit for patients with plasma-cell myeloma because early studies have shown that it may improve the outcome of patients whose disease does not respond to or has come back after other treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Daratumumab is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to a specific structure (an antigen) on the myeloma cells called 'CD38'. Once attached, it is expected to activate the immune system to attack and kill the myeloma cells.
The effects of daratumumab have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with daratumumab in patients with plasma-cell myeloma were ongoing.
At the time of submission, daratumumab was not authorised anywhere in the EU for plasma-cell myeloma. Orphan designation of daratumumab had been granted in the United States for treatment of the condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 13 June 2013 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
- Daratumumab
- Medicine name
- Darzalex
- Intended use
- Treatment of plasma-cell myeloma
- Orphan designation status
- Positive
- EU designation number
- EU/3/13/1153
- Date of designation
- Sponsor
Janssen-Cilag International N.V.
Turnhoutseweg, 30
2340 Beerse
Belgium
Tel. +32 146 031 89
Fax +32 146 055 33
E-mail: http://www.janssen-emea.com/contactus
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of Darzalex at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.
More information is available in the Darzalex : Orphan maintenance assessment report (post-authorisation).
Documents related to this orphan designation evaluation
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:
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.
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: