EU/3/21/2565 - orphan designation for treatment of SCN2A developmental and epileptic encephalopathy

5' moemC-(sp)-moemC-(p)-moeA-(p)-moemC-(p)-moeG-(p)-moeA-(p)-dmC-(sp)-dA-(sp)-dT-(sp)-dA-(sp)-dT-(sp)-dT-(sp)-dT-(sp)-dT-(sp)-dT-(sp)-dmC-(sp)-moeT-(p)-moeA-(sp)-moemC-(sp)-moeA 3'
OrphanHuman

Overview

This medicine was designated as an orphan medicine for the treatment of SCN2A developmental and epileptic encephalopathy 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:

Patients with SCN2A developmental and epileptic encephalopathy have changes in the SCN2A gene, which is involved in the production of certain proteins (voltage-gated sodium channels) located on the nerve cells of the brain, that control transmission of electrical impulses within the brain.

This medicine, also known as PRAX-222, is expected to work by reducing the levels of these sodium channels in nerve cells, by decreasing production of the SCN2A protein, in order to reduce unwanted activity and maintain normal electrical activity in the brain. This is expected to reduce epileptic seizures and improve other neurological symptoms of the disease.

Based on description provided by sponsor

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

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
5' moemC-(sp)-moemC-(p)-moeA-(p)-moemC-(p)-moeG-(p)-moeA-(p)-dmC-(sp)-dA-(sp)-dT-(sp)-dA-(sp)-dT-(sp)-dT-(sp)-dT-(sp)-dT-(sp)-dT-(sp)-dmC-(sp)-moeT-(p)-moeA-(sp)-moemC-(sp)-moeA 3'
Intended use
Treatment of SCN2A developmental and epileptic encephalopathy
Orphan designation status
Positive
EU designation number
EU/3/21/2565
Date of designation
Sponsor

Transcrip Ireland Limited

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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