EU/3/16/1717 - orphan designation for treatment of tracheal stenosis

autologous mesenchymal stromal cells on a decellularised tracheal scaffold from a cadaveric donor

Overview

On 29 August 2016, orphan designation (EU/3/16/1717) was granted by the European Commission to Videregen Ltd, United Kingdom, for autologous mesenchymal stromal cells on a decellularised tracheal scaffold from a cadaveric donor for the treatment of tracheal stenosis.

The sponsorship was transferred to Videregen Europe Limited, Ireland, in June 2019.

What is tracheal stenosis?

Tracheal stenosis is narrowing of the trachea, the windpipe that leads from the throat to the lungs. It can affect both children and adults. The disease can appear at birth or can occur later in life because of injury or certain diseases.

The severity of tracheal stenosis varies depending on the extent of the trachea affected, and symptoms include noisy breathing, shortness of breath and coughing.

Tracheal stenosis is a long-term debilitating and life-threatening condition because it can lead to lung infections and difficulty in swallowing and breathing.

What is the estimated number of patients affected by the condition?

At the time of designation, tracheal stenosis affected less than 2 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 103,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

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*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 513,700,000 (Eurostat 2016).

What treatments are available?

At the time of designation, no medicines were authorised in the EU for the treatment of tracheal stenosis. Some patients underwent surgery to remove damaged areas of the trachea or medical procedures to try to dilate (widen) the trachea.

How is this medicine expected to work?

This medicine is made of:

• a 'tracheal scaffold' from a dead donor, made only of the cartilaginous tube that supports the trachea with all other cells removed;
• cells called 'mesenchymal stromal cells' taken from the patient. These are cells able to develop into a variety of cell types, including osteoblasts (bone cells), chondrocytes (cartilage cells) and adipocytes (fat cells).

The mesenchymal stromal cells are first grown in the laboratory to increase their number and then seeded on the surface of the tracheal scaffold to form a working trachea. The damaged trachea is then removed from the patient and replaced with the seeded tracheal scaffold.

What is the stage of development of this medicine?

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with tracheal stenosis had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for tracheal stenosis or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 13 July 2016 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:

Opinions on orphan medicinal product designations are based on the following three criteria:

• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

EU/3/16/1717 : Public summary of opinion on orphan designation: Autologous mesenchymal stromal cells on a decellularised tracheal scaffold from a cadaveric donor for the treatment of tracheal stenosis

Adopted

Key facts

• Active substance:
  autologous mesenchymal stromal cells on a decellularised tracheal scaffold from a cadaveric donor
• Intended use:
  Treatment of tracheal stenosis
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/16/1717
• Date of designation:
  29/08/2016
• Sponsor:

  Videregen Europe Limited; 1 Grant's Row; Dublin 2; Co. Dublin; D02 HX96; Ireland; Tel. [phone]; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.