EU/3/16/1610 - orphan designation for treatment of acute myeloid leukaemia

arsenic trioxide

Overview

On 17 February 2016, orphan designation (EU/3/16/1610) was granted by the European Commission to Orsenix Holdings BV, the Netherlands, for arsenic trioxide for the treatment of acute myeloid leukaemia.

The sponsorship was transferred to Syros Pharmaceuticals (Ireland) Limited in June 2021.

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 blood cells, causing bleeding episodes, blood clots and 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 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 51,000 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).

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*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 513,700,000 (Eurostat 2016).

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 arsenic trioxide might be of significant benefit for patients with AML because data from the published literature show that it has anti-cancer effects in some patients who have not been treated before. In addition, this medicine can be taken by mouth and so may be easier to take than the currently authorised medicine containing arsenic trioxide (Trisenox) which has to be given into a vein. 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?

Arsenic trioxide is a chemical that has been used in medicines for many years, including for the treatment of leukaemia. The way it works in this disease is not completely understood. It is thought to prevent the production of DNA, which is necessary for leukaemia cells to grow.

What is the stage of development of this medicine?

The effects of this 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 AML were planned.

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.

At the time of submission, the medicine Trisenox (arsenic trioxide given into a vein) was authorised in the EU for the treatment of acute promyelocytic leukaemia (APL), a rare form of AML. Trisenox is for use in patients whose disease had not responded to or had come back after previous treatment.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 21 January 2016 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.

EU/3/16/1610: Public summary of opinion on orphan designation: Arsenic trioxide for the treatment of acute myeloid leukaemia

Adopted Reference Number: EMA/COMP/72409/2016

Key facts

• Active substance:
  arsenic trioxide
• Intended use:
  Treatment of acute myeloid leukaemia
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/16/1610
• Date of designation:
  17/02/2016
• Sponsor:

  Orphix Consulting GmbH;

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.