EU/3/18/2020 - orphan designation for treatment of AL amyloidosis
Daratumumab
OrphanHuman
Overview
Daratumumab has been authorised in the EU as Darzalex since 21 June 2021.
On 25 May 2018, orphan designation (EU/3/18/2020) was granted by the European Commission to Janssen-Cilag International N.V., Belgium, for daratumumab for the treatment of AL amyloidosis.
AL amyloidosis belongs to a group of diseases called systemic amyloidosis in which deposits of proteins (called amyloids) accumulate and cause damage in tissues and organs such as the kidneys, liver, gut, heart and nerves.
In AL amyloidosis, the deposits are made up of proteins (called immunoglobulin light chains) produced in excess by malfunctioning white blood cells in the bone marrow. These deposits also contain a protein normally found in the blood called serum amyloid P.
Symptoms of the condition vary widely depending on which organs are affected by the deposits and how much deposits have accumulated in them.
AL amyloidosis is a life-threatening and long-term debilitating condition because of damage to organs, particularly the heart and kidneys.
At the time of designation, AL amyloidosis affected approximately 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 52,000 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 517,400,000 (Eurostat 2018).
At the time of designation, no medicines were authorised in the EU for the treatment of AL amyloidosis. Patients often received treatment with medicines (chemotherapy) authorised for the treatment of cancers of white blood cells, in order to target the malfunctioning white blood cells.
The medicine is a monoclonal antibody (a type of protein) designed to recognise and attach to a specific structure called 'CD38' which is found in great numbers on the white blood cells that produce immunoglobulin light chains. Once attached, it is expected to activate the immune system to attack and kill the white blood cells. This is expected to reduce the deposits of proteins, and so improve symptoms of the disease.
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 AL amyloidosis were ongoing.
At the time of submission, daratumumab (Darzalex) was authorised in the EU for the treatment of multiple myeloma.
At the time of submission, daratumumab was not authorised anywhere in the EU for AL amyloidosis. Orphan designation of daratumumab had been granted in the United States for this condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 19 April 2018 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
- Intended use
- Treatment of AL amyloidosis
- Orphan designation status
- Positive
- EU designation number
- EU/3/18/2020
- Date of designation
- Sponsor
Janssen-Cilag International N.V.
Turnhoutseweg, 30
2340 Beerse
Belgium
Tel. +32 146 031 89 / +32 14 60 39 73
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 orphan medicine assessment report.
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: