EU/3/22/2698 - orphan designation for treatment of otoferlin gene-mediated hearing loss

Adeno?associated viral vector serotype 8 containing the 3' human otoferlin coding sequence, adeno?associated viral vector serotype 8 containing the 5' human otoferlin coding sequenceAdeno‐associated viral vector serotype 8 containing the 3' human otoferlin coding sequence, adeno‐associated viral vector serotype 8 containing the 5' human otoferlin coding sequence
OrphanHuman

Overview

This medicine was designated as an orphan medicine for the treatment of otoferlin gene-mediated hearing loss in the European Union on 11 October 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:

Otoferlin gene-mediated hearing loss is a rare genetic disease caused by mutations in the OTOF gene, leading to impaired signal transmission to the auditory nerve, and associated hearing loss.

This medicine, also called SENS-501, is used as gene therapy and uses another virus (adenovirus) that has been modified to contain the functioning version of the OTOF gene. The adenovirus in this medicine cannot reproduce and does not cause disease. Treatment with SENS-501 requires ear surgery, where the medicine is injected in a specific area of the ear called the cochlea. By doing so, the mutated OTOF gene is replaced with the functioning version. This procedure is expected to restore hearing in patients with Otoferlin gene-mediated hearing loss.

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 sickle cell disease 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, 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 6-8 September 2022

AdoptedReference Number: EMA/COMP/772376/2022

English (EN) (566.63 KB - PDF)

First published:
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Key facts

Active substance
Adeno?associated viral vector serotype 8 containing the 3' human otoferlin coding sequence, adeno?associated viral vector serotype 8 containing the 5' human otoferlin coding sequenceAdeno‐associated viral vector serotype 8 containing the 3' human otoferlin coding sequence, adeno‐associated viral vector serotype 8 containing the 5' human otoferlin coding sequence
Intended use
Treatment of otoferlin gene-mediated hearing loss
Orphan designation status
Positive
EU designation number
EU/3/22/2698
Date of designation
Sponsor

Sensorion
375 rue du Professeur Blayac
34080 Montpellier
France
E-mail: contact@sensorion-pharma.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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