EU/3/17/1862 - orphan designation for treatment of Asherman's syndrome

autologous adult bone marrow-derived non-expanded CD133+ haematopoietic stem cells

Overview

On 20 April 2017, orphan designation (EU/3/17/1862) was granted by the European Commission to Igenomix, S.L., Spain, for autologous adult bone marrow-derived non-expanded CD133+ haematopoietic stem cells for the treatment of Asherman's syndrome.

The sponsorship was transferred to Asherman Therapy S.L.U., Spain, in March 2020.

What is Asherman's syndrome?

Asherman's syndrome is a condition caused by scarring and sticking together (adhesions) of tissues in the lining and neck of the womb (the endometrium and cervix), often following surgery or the use of an intrauterine device. Adhesions may occur only in a small part of the womb, or be more extensive and severe, causing the front and back walls of the womb to stick to one another. Women with Asherman's syndrome have pain, menstrual cycle (period) irregularities such as reduced or absent menstrual bleeding, frequent miscarriages and infertility.

Asherman's syndrome is a debilitating condition due to the pain, miscarriages and infertility women with the condition experience, and because of the psychological distress associated with it.

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

At the time of designation, Asherman's syndrome affected approximately 4.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 227,000 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 515,700,000 (Eurostat 2017).

What treatments are available?

At the time of designation, treatments for Asherman's syndrome included surgery to remove scar tissue and adhesions, use of devices to prevent the recurrence of adhesions, and hormonal treatment to restore the function of the endometrium.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with Asherman's syndrome because early studies showed that it could improve pregnancy rates in patients affected by the condition in whom other treatments had failed. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

This medicine is made up of the patient's own stem cells. Stem cells are cells that can develop into different types of cell. To make the medicine, a type of stem cells called CD133+ stem cells are collected from the bone marrow until a sufficient number is available.

Although it is not fully understood how they work, when these cells are injected into the blood vessels of the womb they are expected to stimulate the growth of blood vessels in the endometrium. This is expected to facilitate scar healing, thus improving the symptoms of the condition. These stem cells may also be able to develop into cells of the endometrium, thus helping to regenerate the damaged tissue in women with the condition.

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, clinical trials with the medicine in patients with Asherman's syndrome were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for Asherman's syndrome 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 15 March 2017 recommending the granting of this designation.

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/3/17/1862: Public summary of opinion on orphan designation: Autologous adult bone marrow-derived non-expanded CD133+haematopoietic stem cells for the treatment of Asherman's syndrome

Adopted Reference Number: EMA/206895/2017

Key facts

• Active substance:
  autologous adult bone marrow-derived non-expanded CD133+ haematopoietic stem cells
• Intended use:
  Treatment of Asherman's syndrome
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/17/1862
• Date of designation:
  20/04/2017
• Sponsor:

  Asherman Therapy S.L.U.; Calle De Narcis Monturiol I Estarriol 11-B; Parque Tecnologico; 46980 Paterna Valencia; Spain; 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.