OraNera

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Questions and answers

On 14 March 2013, CellSeed Europe Ltd. officially notified the European Medicines Agency that it wishes to withdraw its application for a marketing authorisation for OraNera (previously known as Caomecs), intended for the treatment of limbal-stem-cell deficiency in adults.

What is OraNera?

OraNera is a medicinal product consisting of cells obtained from the lining of the patient’s mouth (autologous oral mucosal epithelial cells). It was to be available as multilayer cell sheets.

OraNera is an advanced-therapy medicine called a ‘tissue-engineered product’. This is a type of medicine that contains cells or tissues that have been ‘engineered’ (modified) so they can be used to repair, regenerate or replace tissue.

What was OraNera expected to be used for?

OraNera was expected to be used to treat limbal-stem-cell deficiency (LSCD) in adults. LSCD is an eye condition in which the patient lacks cells called limbal stem cells, which are found at the edge of the cornea (the transparent layer in front of the eye) and which continuously renew and repair the cornea. The deficiency of limbal stem cells leads to clouding of the cornea and may result in visual impairment or blindness.

How is OraNera expected to work?

One cell sheet of OraNera is expected to be grafted onto the patient’s eye. As patients with LSCD are unable to replace the cells of the cornea, the cells in the OraNera sheet were expected to be used to replace damaged corneal cells, thereby relieving any symptoms the patient may have.

What did the company present to support its application?

The main data submitted by the company came from a main study in 26 patients with LSCD who received OraNera.

How far into the evaluation was the application when it was withdrawn?

The evaluation of advanced therapy products involves an assessment by the Committee for Advanced Therapies (CAT) before an opinion is adopted by the Committee for Medicinal Products for Human Use (CHMP).

This application was withdrawn after the CAT had evaluated the documentation provided by the company and formulated a list of questions. The company had not yet responded to the last round of questions at the time of the withdrawal.

What was the recommendation of the CAT at that time?

At the time of the withdrawal, the CAT had not issued its final recommendation but had concerns about the data submitted in the application.

The CAT was concerned that the data from the main study did not allow any conclusions to be drawn on the beneficial effects of OraNera in patients with LSCD. There were concerns about the quality of the product and the way the study was conducted, such as how treatment success was defined. In addition, the CAT noted that the company would not be able to generate new data necessary to support the current application for marketing authorisation.

Therefore, at the time of the withdrawal, the CAT was of the opinion that the benefits of OraNera did not outweigh its risks.

What were the reasons given by the company for withdrawing the application?

In its letter notifying the Agency of the withdrawal of application, the company state that it was withdrawing its application because, on the basis of the CAT’s preliminary assessment, the Committee could not conclude on a positive benefit-risk balance and the company was unable to generate additional data required for a favourable opinion within the regulatory timeframe.

What consequences does this withdrawal have for patients in clinical trials or compassionate-use programmes?

The company informed the Agency that there are currently no clinical trials or compassionate-use programmes using OraNera.

Name Language First published Last updated
Questions and answers on the withdrawal of the marketing authorisation application for OraNera (English only) 22/03/2013  

Press release

CellSeed Europe Ltd. withdraws its marketing-authorisation application for OraNera (autologous oral mucosal epithelial cells)

The European Medicines Agency has been formally notified by CellSeed Europe Ltd. of its decision to withdraw its application for a centralised marketing authorisation for the medicine OraNera (autologous oral mucosal epithelial cells), a living-tissue equivalent consisting of two autologous oral mucosal epithelial cell sheets. It was intended to be used for treatment of limbal-stem-cell deficiency (LSCD).

The application for the marketing authorisation for OraNera was submitted to the Agency on 1 June 2011. OraNera is an advanced-therapy medicinal product (ATMP). At the time of the withdrawal, the medicine was under review by the Agency’s Committee for Advanced Therapies (CAT) and Committee for Medicinal Products for Human Use (CHMP).

In its official letter, the company stated that it is withdrawing its application because, on the basis of the CAT’s preliminary assessment, the Committee could not conclude on a positive benefit-risk balance. The company is not able to generate additional data required for a favourable opinion within the timeframe agreed in the centralised procedure.

More information about OraNera and the state of the scientific assessment at the time of withdrawal will be made available in a question-and-answer document. This document will be published on the Agency’s website after the CHMP meeting of 18-21 March 2013.

Notes

  • Withdrawal of an application does not prejudice the possibility of a company making a new application at a later stage.
  • Advanced-therapy medicinal products are medicines for human use that are based on gene therapy, somatic-cell therapy or tissue engineering. They offer ground-breaking new opportunities for the treatment of disease and injury.

Key facts

Product details for OraNera
NameOraNera
Product numberEMEA/H/C/002443
Date of issue of marketing authorisation valid throughout the European Union (if applicable)
International non-proprietary name or common name

multilayered cell-sheet of autologous oral mucosal epithelial cells

Active substancemultilayered cell-sheet of autologous oral mucosal epithelial cells
Date of withdrawal14/03/2013
Company making the application

CellSeed Europe Ltd

Withdrawal typeInitial authorisation

All documents

More information on OraNera

Related information on withdrawals

The question-and-answer (Q&A) document provides a summary of the CHMP's evaluation of the medicine at the time of the withdrawal of the application, and includes a link to the company's formal withdrawal letter.

An assessment report is published when the application is withdrawn after the first stage of the CHMP's evaluation is completed ('day 120').