EU/3/13/1174

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Orphan designation

On 5 August 2013, orphan designation (EU/3/13/1174) was granted by the European Commission to Oryzon Genomics SA, Spain, for trans-N1-((1R,2S)-2-phenylcyclopropyl)cyclohexane-1,4-diamine bis-hydrochloride for the treatment of acute myeloid leukaemia.

The sponsorship was transferred to Roche Registration Limited, United Kingdom, in December 2014.

The sponsorship was transferred to Oryzon Genomics SA - Spain, in February 2018. 

What is acute myeloid leukaemia?

Acute myeloid leukaemia (AML) is a cancer of the white blood cells (cells that fight against infections). In patients with AML, the bone marrow (the spongy tissue inside the large bones, where blood cells are produced) produces large numbers of abnormal, immature white blood cells. These abnormal cells quickly build up in large numbers in the bone marrow and are found in the blood.

AML is a long-term debilitating and life-threatening disease because these abnormal immature cells take the place of the normal white blood cells, reducing the patient’s ability to fight infections.

What is the estimated number of patients affected by the condition?

At the time of designation, AML affected approximately 2.7 in 10,000 people in the European Union (EU). This was equivalent to a total of around 138,000 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).

What treatments are available?

Treatment for AML is complex and depends on a number of factors including the extent of the disease, whether it has been treated before, and the patient’s age, symptoms and general state of health. At the time of designation, the main treatments for AML were chemotherapy (medicines to treat cancer) and haematopoietic (blood) stem-cell transplantation (a complex procedure where the patient receives stem cells from a matched donor to help restore the bone marrow).

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with AML because it works in a different way to current treatments, thereby offering an alternative for AML that has come back or does not respond to other treatments, and early studies in experimental models show that it might reduce the growth of the cancer. These assumptions 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 a small molecule that is expected to block the activity of an enzyme called lysine-specific demethylase 1 (LSD1), which is involved in turning genes ‘on’ and ‘off’ within cells. In AML, this medicine is expected to switch ‘on’ the genes that suppress the division and growth of the tumour cells. This is expected to lead to a reduction in the growth of the cancer.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, the evaluation of the effects of the medicine in experimental models was ongoing.

At the time of submission, no clinical trials with the medicine in patients with AML had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for AML or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 11 July 2013 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.

Key facts

Product details for <p>Trans-N1-((1R,2S)-2-phenylcyclopropyl)cyclohexane-1,4-diamine bis-hydrochloride</p>
Active substanceTrans-N1-((1R,2S)-2-phenylcyclopropyl)cyclohexane-1,4-diamine bis-hydrochloride
Medicine Name
Disease/conditionTreatment of acute myeloid leukaemia
Date of decision05/08/2013
OutcomePositive
Orphan decision numberEU/3/13/1174

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

Oryzon Genomics SA
C. Sant Ferran 74
08940Cornella de Llobregat, Barcelona
Spain
Tel. +34 93 515 1313
E-mail: info@oryzon.com

Patients' organisations

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.