EU/3/21/2569 - orphan designation for treatment of acute myeloid leukaemia

trans N-ethyl-2-((4-(7-((4-(ethylsulfonamido)cyclohexyl)methyl)-2,7-diazaspiro[3.5]nonan-2-yl)pyrimidin-5-yl)oxy)-5-fluoro-N-isopropylbenzamide sesquifumarate
OrphanHuman

Overview

This medicine was designated as an orphan medicine for the treatment of acute myeloid leukaemia in the European Union on 14 January 2022.

This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation.

Orphan designation does not mean the medicine is available or authorised for use. All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.

During the medicine's development, doctors may be able to enrol patients in clinical trials investigating the medicine. For information on ongoing clinical trials in the EU, see:

In a normal cell, two proteins called KMT2A and menin collaborate to switch on specific genes. Some patients with acute myeloid leukaemia have mutations (changes) in the KMT2A protein, or changes in another protein called NPM1, which alter the activity of menin. These changes lead to some cancer-causing genes being switched on. This medicine, also known as SNDX-5613, interferes with the interaction between KMT2A and menin, which inhibits activation of these cancer-causing genes and slows the growth of the cancer cells.

Based on description provided by sponsor

At the time of submission of the application for orphan designation:

  • The effects of the medicine had been evaluated in experimental models.
  • Clinical trials with the medicine in patients with acute myeloid leukaemia were ongoing.

More information on how potential new medicines are tested during their development is available on Authorisation of medicines.

Medicines intended for rare diseases can be granted an orphan designation during their development.

The orphan designation allows the developer to benefit from:

  • scientific and regulatory support to advance their medicine to the stage where they can request marketing authorisation;
  • market exclusivity once the medicine is on the market.

To qualify for orphan designation, a medicine must meet a number of criteria:

  • it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
  • the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
  • there are no satisfactory alternative methods for the diagnosis, prevention or treatment of the condition or, if such a method exists, the medicine is of significant benefit to those affected by the condition.

EMA's Committee for Orphan Medicinal Products (COMP) is responsible for issuing opinions on applications for orphan designations.

The Agency sends the COMP opinion to the European Commission, which is responsible for granting the orphan designation. The full list of orphan designations is available in the Community register of orphan medicinal products for human use.

For more information, see:

Minutes of the COMP meeting on 7-9 December 2021

Adopted Reference Number: EMA/COMP/778269/2021

English (EN) (736.47 KB - PDF)

First published:
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Key facts

Active substance
trans N-ethyl-2-((4-(7-((4-(ethylsulfonamido)cyclohexyl)methyl)-2,7-diazaspiro[3.5]nonan-2-yl)pyrimidin-5-yl)oxy)-5-fluoro-N-isopropylbenzamide sesquifumarate
Intended use
Treatment of acute myeloid leukaemia
Orphan designation status
Positive
EU designation number
EU/3/21/2569
Date of designation
Sponsor

Syndax Europe B.V.
Lichttoren 32
5611 BJ Eindhoven
Noord-Brabant
Netherlands
E-mail: info@syndax.com

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:

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