EU/3/15/1567 - orphan designation for treatment of blastic plasmacytoid dendritic cell neoplasm
recombinant human interleukin-3 truncated diphtheria toxin fusion protein
OrphanHuman
Overview
On 11 November 2015, orphan designation (EU/3/15/1567) was granted by the European Commission to Spector Consulting SAS, France, for recombinant human interleukin-3 truncated diphtheria toxin fusion protein (also known as SL-401) for the treatment of blastic plasmacytoid dendritic cell neoplasm.
The sponsorship was transferred to TMC Pharma (EU) Limited, Ireland, in November 2018 and to Stemline Therapeutics B.V. in November 2019.
Recombinant human interleukin-3 truncated diphtheria toxin fusion protein has been authorised in the EU as Elzonris since 7 January 2021.
Blastic plasmacytoid dendritic cell neoplasm is an aggressive type of blood cancer in which the bone marrow (the spongy tissue inside the large bones, where blood cells are produced) produces large numbers of immature white blood cells called 'plasmacytoid dendritic cells'. These may build up in the bone marrow and take the place of normal blood cells. Most patients develop non-itchy damaged areas in the skin, which often look like bruises or nodules. The disease may also cause enlargement of the spleen or liver and a reduction of the number of circulating blood cells.
Blastic plasmacytoid dendritic cell neoplasm is a life-threatening disease that is associated with poor overall survival.
At the time of designation, blastic plasmacytoid dendritic cell neoplasm affected approximately 1.2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 62,000 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 512,900,000 (Eurostat 2015).
At the time of designation, no satisfactory methods were authorised in the EU for the treatment of blastic plasmacytoid dendritic cell neoplasm. Patients often received radiotherapy and medicines authorised for other types of blood cancers. Some patients were also given stem cell transplantation, a complex procedure where the patient receives stem cells from a matched donor to help restore the bone marrow.
This medicine contains a toxin (a substance which is poisonous for cells) called diphtheria toxin, which has been 'fused' to a protein called 'interleukin-3'. Although is it not fully clear how the medicine works, it is expected that the interleukin-3 will attach to interleukin-3 receptors, which are found at high levels on the surface of some cancer cells. Once attached to cancer cells, the medicine is expected to be taken up by the cells. The toxin would then be released inside cancer cells, thus killing them.
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 blastic plasmacytoid dendritic cell neoplasm were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for blastic plasmacytoid dendritic cell neoplasm. Orphan designation of the medicine 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 8 October 2015 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
- recombinant human interleukin-3 truncated diphtheria toxin fusion protein
- Intended use
- Treatment of blastic plasmacytoid dendritic cell neoplasm
- Orphan designation status
- Positive
- EU designation number
- EU/3/15/1567
- Date of designation
- Sponsor
Stemline Therapeutics B.V.
Basisweg 10
1043 AP Amsterdam
Noord-Holland
Netherlands
Tel. + 44 (0)800 047 8675
E-mail: EUmedinfo@menarinistemline.com
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of product at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.
More information is available in the Elzonris : Orphan maintenance assessment report (initial 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:
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: