On 26 April 2012, orphan designation (EU/3/12/987) was granted by the European Commission to JJGConsultancy Ltd, United Kingdom, for (E)-2,4,6-trimethoxystyryl-3-carboxymethylamino-4-methoxybenzyl-sulfone sodium salt for the treatment of myelodysplastic syndromes.
The sponsorship was transferred to Onconova Europe GmbH, Germany, in February 2014.
- What are myelodysplastic syndromes?
Myelodysplastic syndromes (MDSs) are a group of disorders in which the red blood cells, white blood cells and platelets produced by the bone marrow (the spongy tissue inside the large bones) do not grow and mature normally. Patients with MDSs can develop several symptoms including tiredness or weakness due to anaemia (low red blood cell counts), infections due to low white blood cells, and bruising or abnormal bleeding due to low platelet counts.
MDSs are long-term debilitating and life-threatening diseases because they can lead to severe anaemia, infections or bleeding, and can result in leukaemia (cancer of the white blood cells).
- What is the estimated number of patients affected by the condition?
At the time of designation, MDSs affected not more 2 in 10,000 people in the European Union (EU)*. This is equivalent to a total of not more than 102,000 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 27), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 509,000,000 (Eurostat 2012).
- What treatments are available?
At the time of designation, some medicines were authorised in the EU for the treatment of MDSs. The choice of treatment for MDSs depended on a number of factors, including the type and the extent of the disease, whether it had been treated before, and the patient’s age, symptoms and general state of health. The main treatments for MDSs included chemotherapy (medicines to treat cancer) and bone marrow transplantation. This is a complex procedure where the bone marrow of the patient is cleared of cells and replaced with healthy bone marrow cells from a matched donor.
The sponsor has provided sufficient information to show that (E)-2,4,6-trimethoxystyryl-3-carboxymethylamino-4-methoxybenzyl-sulfone sodium salt might be of significant benefit for patients with MDSs because early studies show that it might improve the treatment of patients who have not responded to previous treatment, when used alone or in combination with other authorised medicines. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
- How is this medicine expected to work?
This medicine is thought to work by blocking different types of enzymes known as protein kinases, which are involved in the growth and division of cancer cells. By blocking these enzymes, this medicine is expected to block the division of the abnormal blood cells in MDSs, leading to cell death.
- What is the stage of development of this medicine?
The effects of (E)-2,4,6-trimethoxystyryl-3-carboxymethylamino-4-methoxybenzyl-sulfone sodium salt have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with this medicine in patients with MDSs were ongoing.
At the time of submission, this medicine was not authorised anywhere in the EU for MDSs. Orphan designation of this medicine had been granted in the United States of America for MDSs.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 March 2012 recommending the granting of this designation.
- Opinions on orphan medicinal product designations are based on the following three criteria:
- 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.
|Name||Language||First published||Last updated|
|EU/3/12/987: Public summary of opinion on orphan designation: (E)-2,4,6-trimethoxystyryl-3-carboxymethylamino-4-methoxybenzyl-sulfone sodium salt for the treatment of myelodysplastic syndromes||(English only)||2012-06-18||2014-05-08|
|Active substance||(E)-2,4,6-trimethoxystyryl-3-carboxymethylamino-4-methoxybenzyl-sulfone sodium salt|
|Disease/condition||Treatment of myelodysplastic syndromes|
|Date of decision||26/04/2012|
|Orphan decision number||EU/3/12/987|
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
Sponsor’s contact details
Onconova Europe GmbH
Straßl ins Holz 13
Tel. +49 172 860 8632
Fax +49 (0) 89 431 1114
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.