EU/3/21/2474 - orphan designation for treatment of neuronal ceroid lipofuscinosis

autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector encoding for the human palmitoyl-protein thioesterase 1 gene
OrphanHuman

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Overview

This medicine was designated as an orphan medicine for the treatment of neuronal ceroid lipofuscinosis in the European Union on 19 July 2021.

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:

This medicine is expected to be used in patients with neuronal ceroid lipofuscinosis type 1. These patients have mutations (changes) in the CLN1 gene that is responsible for the production of an enzyme called PPT1 needed for proper breakdown of molecules called lipofuscins in the body.

The medicine consists of blood stem cells taken from the patient and treated with a modified virus containing the gene for the missing PPT1 enzyme. When the stem cells are given back to the patient, the cells will produce the missing enzyme, breaking down the lipofuscins and thereby helping to relieve symptoms of the disease. The virus used in this medicine does not cause disease in humans.

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.
  • No clinical trials with the medicine in patients with neuronal ceroid lipofuscinosis had been started.

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 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 15-17 June 2021

Adopted Reference Number: EMA/COMP/359170/2021

English (EN) (551.28 KB - PDF)

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Key facts

Active substance
autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector encoding for the human palmitoyl-protein thioesterase 1 gene
Intended use
Treatment of neuronal ceroid lipofuscinosis
Orphan designation status
Positive
EU designation number
EU/3/21/2474
Date of designation
Sponsor

University of Padua
Via Nicolo' Giustiniani 3
35128 Padova (PD)
Italy
Tel. +39 0498218030
E-mail: alessandra.biffi@unipd.it

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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